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A Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent Chronic Kidney Disease (CKD) Patients With Hyperphosphataemia

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT02688764
Recruitment Status : Terminated (The study was prematurely ended due to the modification of study requirements by the US Food and Drug Administration and the European Medicines Agency)
First Posted : February 23, 2016
Results First Posted : August 28, 2019
Last Update Posted : September 10, 2019
Sponsor:
Information provided by (Responsible Party):
Vifor Fresenius Medical Care Renal Pharma

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: None (Open Label);   Primary Purpose: Treatment
Condition Hyperphosphatemia
Interventions Drug: PA21 (Velphoro®)
Drug: Calcium Acetate (Phoslyra®)
Enrollment 85
Recruitment Details  
Pre-assignment Details  
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Period Title: Overall Study
Started 66 19
Treated in Stage 1 66 19
Treated in Stage 2 43 8
Completed 26 2
Not Completed 40 17
Reason Not Completed
Adverse Event             3             3
AE/Kidney transplant             1             0
AE/Lack of efficacy             1             0
AE/Non-compl.             1             0
AE/Non-compl./Withd. parent             0             1
AE/Other             0             1
AE/Physician decision             1             0
AE/Withd. parent             3             0
AE/Withd. parent/Withd. subject             1             1
AE/Withd. subject             1             0
Kidney transplant             10             4
Lack of Efficacy             3             2
Lack of efficacy/Physician decision             0             1
Non-compliance with study drug             4             0
Non-compl./Physician decision             2             0
Non-compl./Ph. decision/Withd. subject             1             1
Non-compl./Withd. subject             0             2
Phosphorus level outside safe range             0             1
Phosphorus >7 mg/dL - Withd. by sponsor             1             0
Study drug taste/Withd. parent             1             0
Subject moving out             2             0
Subject taken off dialysis             1             0
Withdrawal by parent             3             0
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®) Total
Hide Arm/Group Description

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Total of all reporting groups
Overall Number of Baseline Participants 66 19 85
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 66 participants 19 participants 85 participants
12.2  (4.07) 12.6  (3.73) 12.3  (3.98)
Age, Customized  
Measure Type: Count of Participants
Unit of measure:  Participants
Age, Categorical Number Analyzed 66 participants 19 participants 85 participants
Newborns (0-27 days)
0
   0.0%
0
   0.0%
0
   0.0%
Infants (28 days-23 months)
0
   0.0%
0
   0.0%
0
   0.0%
Children (2-11 years)
23
  34.8%
6
  31.6%
29
  34.1%
Adolescents (12-17 years)
42
  63.6%
13
  68.4%
55
  64.7%
Adults (18-64 years)
1
   1.5%
0
   0.0%
1
   1.2%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 66 participants 19 participants 85 participants
Female
34
  51.5%
13
  68.4%
47
  55.3%
Male
32
  48.5%
6
  31.6%
38
  44.7%
Ethnicity (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 66 participants 19 participants 85 participants
Hispanic or Latino
20
  30.3%
5
  26.3%
25
  29.4%
Not Hispanic or Latino
46
  69.7%
14
  73.7%
60
  70.6%
Unknown or Not Reported
0
   0.0%
0
   0.0%
0
   0.0%
Race (NIH/OMB)  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 66 participants 19 participants 85 participants
American Indian or Alaska Native
1
   1.5%
0
   0.0%
1
   1.2%
Asian
0
   0.0%
0
   0.0%
0
   0.0%
Native Hawaiian or Other Pacific Islander
1
   1.5%
1
   5.3%
2
   2.4%
Black or African American
9
  13.6%
2
  10.5%
11
  12.9%
White
43
  65.2%
14
  73.7%
57
  67.1%
More than one race
0
   0.0%
0
   0.0%
0
   0.0%
Unknown or Not Reported
12
  18.2%
2
  10.5%
14
  16.5%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 66 participants 19 participants 85 participants
United States 39 14 53
France 6 0 6
Lithuania 3 0 3
Poland 4 2 6
Germany 5 1 6
Russia 1 1 2
Romania 8 1 9
Height  
Mean (Standard Deviation)
Unit of measure:  Cm
Number Analyzed 66 participants 19 participants 85 participants
142.3  (24.10) 140.3  (24.12) 141.9  (23.98)
Weight  
Mean (Standard Deviation)
Unit of measure:  Kg
Number Analyzed 66 participants 19 participants 85 participants
41.90  (18.023) 38.28  (17.834) 41.10  (17.939)
Body Mass Index  
Mean (Standard Deviation)
Unit of measure:  Kg/m^2
Number Analyzed 66 participants 19 participants 85 participants
19.621  (4.6182) 18.483  (4.4238) 19.366  (4.5743)
1.Primary Outcome
Title Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the PA21 Group
Hide Description [Not Specified]
Time Frame From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) Population: all participants randomised to treatment at Stage 1 who received at least 1 dose of randomised treatment and who had at least 1 post-baseline assessment of the efficacy endpoint (serum phosphorus level), analysed according to treatment randomised.
Arm/Group Title PA21 (Velphoro®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 65
Least Squares Mean (Standard Error)
Unit of Measure: mmol/L
-0.120  (0.081)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PA21 (Velphoro®)
Comments Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 1 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.1465
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
2.Primary Outcome
Title Number and Percentage of Participants Who Withdrew Due to Treatment Emergent Adverse Events
Hide Description Any adverse event Leading to Study Drug Withdrawal is considered.
Time Frame through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Measure Type: Count of Participants
Unit of Measure: Participants
12
  18.2%
6
  31.6%
3.Primary Outcome
Title Number and Percentage of Participants With Any Treatment Emergent Adverse Event
Hide Description

Please note that in this section we are presenting just the overview of the adverse events experienced by the trial participants, in particular, the number of participants with at least one TEAEs until end of stage 2.

Please refer to the detailed tables included on the Adverse Event Module for specifics.

Time Frame through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Measure Type: Count of Participants
Unit of Measure: Participants
50
  75.8%
14
  73.7%
4.Secondary Outcome
Title Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the Phoslyra Group
Hide Description [Not Specified]
Time Frame From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) Population: all participants randomised to treatment at Stage 1 who received at least 1 dose of randomised treatment and who had at least 1 post-baseline assessment of the efficacy endpoint (serum phosphorus level), analysed according to treatment randomised.
Arm/Group Title Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 15
Least Squares Mean (Standard Error)
Unit of Measure: mmol/L
-0.615  (0.320)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Calcium Acetate (Phoslyra®)
Comments Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 1 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0872
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
5.Secondary Outcome
Title Change in Serum Phosphorus Level From Baseline to the End of Stage 2 in Both Groups
Hide Description [Not Specified]
Time Frame From baseline to study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) Population: all participants randomised to treatment at Stage 1 who received at least 1 dose of randomised treatment and who had at least 1 post-baseline assessment of the efficacy endpoint (serum phosphorus level), analysed according to treatment randomised.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 36 6
Least Squares Mean (Standard Error)
Unit of Measure: mmol/L
0.099  (0.198) -0.393  (0.218)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PA21 (Velphoro®)
Comments Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 2 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.6207
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection Calcium Acetate (Phoslyra®)
Comments Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 2 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects.
Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.3226
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
6.Secondary Outcome
Title Participants With Serum Phosphorus Levels Within the Age-dependent Target Range in Each Stage
Hide Description

Number and percentages of participants with serum phosphorus levels below, within and above age-dependent target ranges at baseline, at the end of Stage 1 and at the end of Stage 2.

The age target ranges for serum phosphorus levels are:

  • 0 to <1 year 1.62-2.52 mmol/L
  • 1 year to <6 years 1.45-2.10 mmol/L
  • 6 years to <13 years 1.16-1.87 mmol/L
  • 13 years to ≤18 years 0.74-1.45 mmol/L
Time Frame through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) Population: all participants randomised to treatment at Stage 1 who received at least 1 dose of randomised treatment and who had at least 1 post-baseline assessment of the efficacy endpoint (serum phosphorus level), analysed according to treatment randomised.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 65 15
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Number Analyzed 65 participants 15 participants
Below
2
   3.1%
1
   6.7%
Within
11
  16.9%
1
   6.7%
Above
52
  80.0%
13
  86.7%
End of Stage 1 Number Analyzed 64 participants 15 participants
Below
2
   3.1%
1
   6.7%
Within
25
  39.1%
2
  13.3%
Above
37
  57.8%
12
  80.0%
End of Stage 2 Number Analyzed 40 participants 8 participants
Below
1
   2.5%
0
   0.0%
Within
14
  35.0%
2
  25.0%
Above
25
  62.5%
6
  75.0%
7.Secondary Outcome
Title Participants With Serum Phosphorus Levels Within the Age Related Normal Range in Each Stage
Hide Description

Number and percentages of participants with serum phosphorus levels below, within and above age-dependent normal ranges at baseline, at the end of Stage 1 and at the end of Stage 2.

The age related normal ranges for serum phosphorus levels are:

  • 0 to <1year 1.36 - 2.62 mmol/L
  • 1 year to <6 years 1.03 - 1.97 mmol/L
  • 6 years to <9 years 1.03 - 1.97 mmol/L
  • 9 years to <10 years 1.03 - 1.97 mmol/L
  • 10 years to <15 years 1.00 - 1.94 mmol/L
  • 15 years to ≤18 years 0.71 - 1.65 mmol/L
Time Frame through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) Population: all participants randomised to treatment at Stage 1 who received at least 1 dose of randomised treatment and who had at least 1 post-baseline assessment of the efficacy endpoint (serum phosphorus level), analysed according to treatment randomised.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 65 15
Measure Type: Count of Participants
Unit of Measure: Participants
Baseline Number Analyzed 65 participants 15 participants
Below
1
   1.5%
1
   6.7%
Within
24
  36.9%
5
  33.3%
Above
40
  61.5%
9
  60.0%
End of Stage 1 Number Analyzed 64 participants 15 participants
Below
1
   1.6%
1
   6.7%
Within
39
  60.9%
6
  40.0%
Above
24
  37.5%
8
  53.3%
End of Stage 2 Number Analyzed 40 participants 8 participants
Below
1
   2.5%
0
   0.0%
Within
23
  57.5%
2
  25.0%
Above
16
  40.0%
6
  75.0%
8.Secondary Outcome
Title Serum Phosphorus Values at Each Visit
Hide Description [Not Specified]
Time Frame through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) Population: all participants randomised to treatment at Stage 1 who received at least 1 dose of randomised treatment and who had at least 1 post-baseline assessment of the efficacy endpoint (serum phosphorus level), analysed according to treatment randomised.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 65 15
Mean (Standard Deviation)
Unit of Measure: mmol/L
Baseline Number Analyzed 65 participants 15 participants
2.07  (0.522) 2.15  (0.669)
End of Stage 1 Number Analyzed 64 participants 15 participants
1.82  (0.575) 2.17  (0.804)
End of Stage 2 Number Analyzed 40 participants 8 participants
1.71  (0.506) 2.09  (0.594)
9.Secondary Outcome
Title Serum Total Corrected Calcium at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: mmol/L
Baseline Number Analyzed 66 participants 19 participants
2.38  (0.144) 2.37  (0.185)
End of Stage 1 - Observed data Number Analyzed 65 participants 17 participants
2.35  (0.212) 2.31  (0.130)
End of Stage 2 - Observed data Number Analyzed 41 participants 8 participants
2.28  (0.185) 2.36  (0.154)
End of Stage 1 - Change from baseline Number Analyzed 65 participants 17 participants
-0.03  (0.224) -0.06  (0.212)
End of Stage 2 - Change from baseline Number Analyzed 41 participants 8 participants
-0.06  (0.197) -0.07  (0.207)
10.Secondary Outcome
Title Participants With Sustained Hypercalcaemia
Hide Description Number and percentages of participants with at least 1 episode of sustained hypercalcaemia (defined as total calcium value above the upper safety limit confirmed by repeat sample 1 week later) during the study
Time Frame through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Measure Type: Count of Participants
Unit of Measure: Participants
6
   9.1%
4
  21.1%
11.Secondary Outcome
Title Serum Total Corrected Calcium-Phosphorus Product at Each Time Point and Change From Baseline
Hide Description Summary statistics of Serum total corrected calcium-phosphorus product at each time point and change from baseline, where serum total corrected calcium-phosphorus product correspond to the product of serum total calcium and Phosphorus, expressed in mmol^2/L^2
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: mmol^2/L^2
Baseline Number Analyzed 66 participants 19 participants
4.94  (1.400) 5.31  (1.831)
End of Stage 1 - Observed data Number Analyzed 65 participants 17 participants
4.25  (1.392) 5.05  (1.608)
End of Stage 2 - Observed data Number Analyzed 40 participants 8 participants
3.91  (1.211) 4.90  (1.289)
End of Stage 1 - Change from baseline Number Analyzed 65 participants 17 participants
-0.65  (1.221) -0.31  (2.152)
End of Stage 2 - Change from baseline Number Analyzed 40 participants 8 participants
-0.49  (1.384) -0.12  (1.227)
12.Secondary Outcome
Title Serum iPTH Levels at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: pmol/L
Baseline Number Analyzed 65 participants 19 participants
30.36  (26.393) 37.12  (19.527)
End of Stage 1 - Observed data Number Analyzed 64 participants 15 participants
28.28  (20.972) 45.90  (28.363)
End of Stage 2 - Observed data Number Analyzed 41 participants 8 participants
35.60  (32.903) 52.30  (36.143)
End of Stage 1 - Change from baseline Number Analyzed 64 participants 15 participants
-2.49  (22.139) 5.72  (20.166)
End of Stage 2 - Change from baseline Number Analyzed 40 participants 8 participants
4.61  (38.858) 12.01  (27.151)
13.Secondary Outcome
Title Ferritin Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: ug/L
Baseline Number Analyzed 56 participants 16 participants
223.67  (274.071) 234.28  (228.825)
End of Stage 1 - Observed data Number Analyzed 58 participants 15 participants
322.59  (368.823) 310.46  (369.869)
End of Stage 2 - Observed data Number Analyzed 33 participants 8 participants
326.13  (304.864) 345.13  (366.230)
End of Stage 1 - Change from baseline Number Analyzed 51 participants 14 participants
48.59  (168.999) 59.27  (219.660)
End of Stage 2 - Change from baseline Number Analyzed 28 participants 8 participants
137.57  (149.176) 110.69  (211.054)
14.Secondary Outcome
Title Unsaturated Iron Binding Capacity Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: umol/L
Baseline Number Analyzed 65 participants 19 participants
28.68  (12.985) 25.54  (14.153)
End of Stage 1 - Observed data Number Analyzed 64 participants 16 participants
25.37  (11.162) 27.28  (9.671)
End of Stage 2 - Observed data Number Analyzed 40 participants 8 participants
25.53  (11.672) 26.04  (11.088)
End of Stage 1 - Change from baseline Number Analyzed 63 participants 16 participants
-3.64  (9.284) -0.81  (7.176)
End of Stage 2 - Change from baseline Number Analyzed 39 participants 8 participants
-4.38  (12.454) -3.53  (11.779)
15.Secondary Outcome
Title Iron Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: umol/L
Baseline Number Analyzed 65 participants 19 participants
13.20  (6.948) 15.25  (8.488)
End of Stage 1 - Observed data Number Analyzed 64 participants 16 participants
14.70  (7.425) 12.66  (4.821)
End of Stage 2 - Observed data Number Analyzed 40 participants 8 participants
14.31  (6.005) 13.08  (4.967)
End of Stage 1 - Change from baseline Number Analyzed 63 participants 16 participants
1.64  (8.946) -0.18  (7.197)
End of Stage 2 - Change from baseline Number Analyzed 39 participants 8 participants
1.95  (6.940) 1.35  (9.691)
16.Secondary Outcome
Title Transferrin Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: g/L
Baseline Number Analyzed 66 participants 19 participants
2.12  (0.607) 1.99  (0.565)
End of Stage 1 - Observed data Number Analyzed 65 participants 16 participants
1.96  (0.520) 1.97  (0.588)
End of Stage 2 - Observed data Number Analyzed 41 participants 8 participants
1.89  (0.513) 1.98  (0.439)
End of Stage 1 - Change from baseline Number Analyzed 65 participants 16 participants
-0.17  (0.342) -0.05  (0.234)
End of Stage 2 - Change from baseline Number Analyzed 41 participants 8 participants
-0.28  (0.506) -0.12  (0.289)
17.Secondary Outcome
Title 25-Hydroxy Vitamin D Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: nmol/L
Baseline Number Analyzed 56 participants 17 participants
83.1  (38.74) 81.7  (40.32)
End of Stage 1 - Observed data Number Analyzed 31 participants 10 participants
79.8  (34.25) 74.4  (38.68)
End of Stage 2 - Observed data Number Analyzed 32 participants 8 participants
76.0  (37.48) 59.0  (17.18)
End of Stage 1 - Change from baseline Number Analyzed 26 participants 10 participants
-4.1  (24.28) -9.7  (22.68)
End of Stage 2 - Change from baseline Number Analyzed 27 participants 8 participants
-18.3  (27.77) -21.8  (15.28)
18.Secondary Outcome
Title Bone Specific Alkaline Phosphatase Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: ug/L
Baseline Number Analyzed 55 participants 17 participants
47.77  (35.469) 55.55  (50.249)
End of Stage 1 - Observed data Number Analyzed 31 participants 10 participants
50.37  (42.382) 56.15  (64.537)
End of Stage 2 - Observed data Number Analyzed 32 participants 8 participants
55.31  (43.363) 71.46  (64.427)
End of Stage 1 - Change from baseline Number Analyzed 26 participants 10 participants
7.29  (28.837) 2.38  (27.375)
End of Stage 2 - Change from baseline Number Analyzed 26 participants 8 participants
7.74  (41.156) -4.18  (38.713)
19.Secondary Outcome
Title Type I Collagen C-Telopeptides Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: ug/L
Baseline Number Analyzed 54 participants 17 participants
4.77  (1.201) 4.31  (1.549)
End of Stage 1 - Observed data Number Analyzed 32 participants 9 participants
4.57  (1.549) 5.10  (1.799)
End of Stage 2 - Observed data Number Analyzed 29 participants 7 participants
4.21  (1.591) 4.43  (1.565)
End of Stage 1 - Change from baseline Number Analyzed 26 participants 9 participants
-0.45  (1.387) -0.03  (1.217)
End of Stage 2 - Change from baseline Number Analyzed 23 participants 7 participants
-0.42  (1.893) -0.34  (0.970)
20.Secondary Outcome
Title Fibroblast Growth Factor 23 Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: pg/mL
Baseline Number Analyzed 53 participants 14 participants
820.1  (503.90) 814.9  (468.89)
End of Stage 1 - Observed data Number Analyzed 29 participants 8 participants
775.7  (514.86) 767.7  (482.47)
End of Stage 2 - Observed data Number Analyzed 32 participants 6 participants
603.5  (531.86) 747.4  (628.27)
End of Stage 1 - Change from baseline Number Analyzed 23 participants 7 participants
-60.7  (454.95) -41.8  (729.27)
End of Stage 2 - Change from baseline Number Analyzed 27 participants 5 participants
-187.6  (507.26) 47.8  (467.87)
21.Secondary Outcome
Title Osteocalcin-CL Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: ug/L
Baseline Number Analyzed 54 participants 17 participants
517.9  (357.11) 681.5  (445.94)
End of Stage 1 - Observed data Number Analyzed 29 participants 9 participants
586.0  (421.72) 753.1  (412.38)
End of Stage 2 - Observed data Number Analyzed 32 participants 8 participants
516.9  (429.24) 709.0  (462.59)
End of Stage 1 - Change from baseline Number Analyzed 23 participants 9 participants
28.4  (248.44) 20.0  (179.26)
End of Stage 2 - Change from baseline Number Analyzed 27 participants 8 participants
20.3  (342.77) -91.0  (200.09)
22.Secondary Outcome
Title Tartrate-resistant Acid Phosphatase 5b Values at Each Time Point and Change From Baseline
Hide Description [Not Specified]
Time Frame From baseline through study completion, up to 34 weeks after treatment start date
Hide Outcome Measure Data
Hide Analysis Population Description
Safety Population: all participants who received at least 1 dose of study medication, analysed according to treatment received.
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron

PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 66 19
Mean (Standard Deviation)
Unit of Measure: U/L
Baseline Number Analyzed 50 participants 15 participants
8.86  (7.646) 8.82  (5.161)
End of Stage 1 - Observed data Number Analyzed 30 participants 9 participants
8.55  (8.305) 8.82  (5.160)
End of Stage 2 - Observed data Number Analyzed 29 participants 5 participants
8.25  (5.643) 7.62  (2.044)
End of Stage 1 - Change from baseline Number Analyzed 23 participants 8 participants
0.02  (2.746) -0.11  (4.580)
End of Stage 2 - Change from baseline Number Analyzed 23 participants 5 participants
0.72  (4.108) -0.16  (2.422)
23.Post-Hoc Outcome
Title Change in Serum Phosphorus Level From Baseline to End of Stage 1 in PA21 Group, by Age Group
Hide Description [Not Specified]
Time Frame From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) Population: all participants randomised to treatment at Stage 1 who received at least 1 dose of randomised treatment and who had at least 1 post-baseline assessment of the efficacy endpoint (serum phosphorus level), analysed according to treatment randomised.
Arm/Group Title PA21 (Velphoro®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 65
Least Squares Mean (Standard Error)
Unit of Measure: mmol/L
<2 years Number Analyzed 0 participants
>=2 years to <6 years Number Analyzed 6 participants
-0.078  (0.123)
>=6 years to <12 years Number Analyzed 17 participants
-0.200  (0.158)
>=12 years to <=18 years Number Analyzed 42 participants
-0.149  (0.062)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PA21 (Velphoro®)
Comments

Age group of >=2 years to <6 years

Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 1 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects.

Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5682
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection PA21 (Velphoro®)
Comments

Age group of >=6 years to <12 years

Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 1 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects.

Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.2271
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
Hide Statistical Analysis 3
Statistical Analysis Overview Comparison Group Selection PA21 (Velphoro®)
Comments

Age group of >=12 years to <=18 years

Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 1 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects.

Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0220
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
24.Post-Hoc Outcome
Title Change in Serum Phosphorus (SP) Level From Baseline to End of Stage 1 in PA21 Group, by Serum Phosphorus Level at Baseline
Hide Description The levels of Serum Phosphorus considered at baseline are those above vs within/below Age Related Normal Range
Time Frame From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date)
Hide Outcome Measure Data
Hide Analysis Population Description
Full Analysis Set (FAS) Population: all participants randomised to treatment at Stage 1 who received at least 1 dose of randomised treatment and who had at least 1 post-baseline assessment of the efficacy endpoint (serum phosphorus level), analysed according to treatment randomised.
Arm/Group Title PA21 (Velphoro®)
Hide Arm/Group Description:

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Overall Number of Participants Analyzed 65
Least Squares Mean (Standard Error)
Unit of Measure: mmol/L
SP above Age Related Normal Range Number Analyzed 40 participants
-0.282  (0.096)
SP below/within Related Normal Range Number Analyzed 25 participants
0.082  (0.146)
Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection PA21 (Velphoro®)
Comments

Group of SP at baseline above Age Related Normal Range

Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 1 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects.

Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.0058
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
Hide Statistical Analysis 2
Statistical Analysis Overview Comparison Group Selection PA21 (Velphoro®)
Comments

Group of SP at baseline below or within Age Related Normal Range

Results are obtained from a linear mixed model which includes change in serum phosphorus levels from baseline to the end of Stage 1 as dependent variable and treatment, baseline serum phosphorus, age (in categories) at randomisation, region (Non-US/US) and gender as fixed effects.

Type of Statistical Test Superiority
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.5801
Comments 0.05 level of significance
Method t-test, 2 sided
Comments [Not Specified]
Time Frame Through study completion, up to 34 weeks after treatment start date
Adverse Event Reporting Description TEAEs (treatment emergent adverse events) are included in this section.
 
Arm/Group Title PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Hide Arm/Group Description

Formulations:

PA21 (Velphoro®), chewable tablets 500 mg and 250 mg iron PA21 (Velphoro®), powder for oral suspension 500 mg, 250 mg and 125 mg iron

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's age. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

Formulation:

Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.

Stage 1 (Open-Label Dose Titration; up to 10 weeks): starting dose based on the participant's weight or, if considered more appropriate by the Investigator, at an equivalent dose of their previous phosphate binder (PB), calcium-based or sevelamer. Dose was increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

Stage 2 (Open-Label Safety Extension, 24 week safety extension): dose received at the end of Stage 1, unless a dose change is required. Doses could be titrated for efficacy (to achieve age specific target Serum phosphorus levels), provided a subject had been receiving that dose for a minimum of 2 weeks, and for safety/tolerability reasons at any time.

All-Cause Mortality
PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Affected / at Risk (%) Affected / at Risk (%)
Total   0/66 (0.00%)      0/19 (0.00%)    
Hide Serious Adverse Events
PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   18/66 (27.27%)      3/19 (15.79%)    
Cardiac disorders     
Bradycardia * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Cardiac tamponade * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Eye disorders     
Papilloedema * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Gastrointestinal disorders     
Gastritis * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Ileus * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Small intestinal obstruction * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Small intestinal perforation * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Vomiting * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
General disorders     
Catheter site haematoma * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Oedema peripheral * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Puncture site reaction * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Pyrexia * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Infections and infestations     
Device related sepsis * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Sepsis * 1  1/66 (1.52%)  2 0/19 (0.00%)  0
Superinfection * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Tonsillitis streptococcal * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Injury, poisoning and procedural complications     
Arteriovenous fistula site complication * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Arteriovenous fistula site haematoma * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Arteriovenous fistula thrombosis * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Investigations     
Weight increased * 1  2/66 (3.03%)  2 0/19 (0.00%)  0
Blood creatinine increased * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Blood pressure increased * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Glomerular filtration rate decreased * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Weight decreased * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Metabolism and nutrition disorders     
Fluid overload * 1  2/66 (3.03%)  2 0/19 (0.00%)  0
Decreased appetite * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Dehydration * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Electrolyte imbalance * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Nervous system disorders     
Benign intracranial hypertension * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Product Issues     
Device malfunction * 1  2/66 (3.03%)  3 0/19 (0.00%)  0
Device extrusion * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Device occlusion * 1  0/66 (0.00%)  0 1/19 (5.26%)  2
Renal and urinary disorders     
Azotaemia * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
End stage renal disease * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Hydronephrosis * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Respiratory, thoracic and mediastinal disorders     
Lung disorder * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Vascular disorders     
Hypertension * 1  5/66 (7.58%)  6 0/19 (0.00%)  0
Hypotension * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Vena cava thrombosis * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Venous thrombosis * 1  1/66 (1.52%)  1 0/19 (0.00%)  0
Malignant hypertension * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
1
Term from vocabulary, MedDRA 19.1
*
Indicates events were collected by non-systematic assessment
Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
PA21 (Velphoro®) Calcium Acetate (Phoslyra®)
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   45/66 (68.18%)      14/19 (73.68%)    
Ear and labyrinth disorders     
Otorrhoea * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Endocrine disorders     
Hyperparathyroidism * 1  2/66 (3.03%)  3 1/19 (5.26%)  1
Hyperparathyroidism secondary * 1  1/66 (1.52%)  2 1/19 (5.26%)  1
Gastrointestinal disorders     
Diarrhoea * 1  12/66 (18.18%)  14 0/19 (0.00%)  0
Nausea * 1  8/66 (12.12%)  10 2/19 (10.53%)  2
Vomiting * 1  6/66 (9.09%)  6 2/19 (10.53%)  3
Constipation * 1  4/66 (6.06%)  4 1/19 (5.26%)  1
Abdominal pain * 1  3/66 (4.55%)  3 1/19 (5.26%)  1
Abdominal pain upper * 1  2/66 (3.03%)  2 1/19 (5.26%)  1
General disorders     
Pyrexia * 1  3/66 (4.55%)  3 1/19 (5.26%)  1
Catheter site haemorrhage * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Infections and infestations     
Urinary tract infection * 1  3/66 (4.55%)  5 2/19 (10.53%)  3
Upper respiratory tract infection * 1  2/66 (3.03%)  2 1/19 (5.26%)  2
Gastroenteritis * 1  1/66 (1.52%)  1 1/19 (5.26%)  1
Pharyngitis * 1  1/66 (1.52%)  1 1/19 (5.26%)  1
Clostridium difficile colitis * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Clostridium difficile infection * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Conjunctivitis * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Cystitis * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Hand-foot-and-mouth disease * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Respiratory syncytial virus infection * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Staphylococcal bacteraemia * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Investigations     
Blood lactate dehydrogenase increased * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Blood phosphorus increased * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Liver function test increased * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Metabolism and nutrition disorders     
Hypercalcaemia * 1  4/66 (6.06%)  4 4/19 (21.05%)  4
Hyperphosphataemia * 1  3/66 (4.55%)  3 3/19 (15.79%)  3
Hyperkalaemia * 1  2/66 (3.03%)  3 1/19 (5.26%)  1
Hypophosphataemia * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Iron deficiency * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Metabolic acidosis * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Nervous system disorders     
Headache * 1  2/66 (3.03%)  2 1/19 (5.26%)  1
Dizziness * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Renal and urinary disorders     
Dysuria * 1  1/66 (1.52%)  1 1/19 (5.26%)  1
Haematuria * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Reproductive system and breast disorders     
Amenorrhoea * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Respiratory, thoracic and mediastinal disorders     
Cough * 1  1/66 (1.52%)  1 2/19 (10.53%)  2
Sinus congestion * 1  0/66 (0.00%)  0 1/19 (5.26%)  2
Skin and subcutaneous tissue disorders     
Excessive granulation tissue * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Pruritus * 1  0/66 (0.00%)  0 1/19 (5.26%)  1
Rash * 1  0/66 (0.00%)  0 1/19 (5.26%)  2
1
Term from vocabulary, MedDRA 19.1
*
Indicates events were collected by non-systematic assessment
The study was prematurely ended as a result of the modification of study requirements, as agreed with the US Food and Drug Administration (FDA) and the European Medicines Agency (EMA).
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There IS an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.
No results of the Services or other information on the Clinical Study may be disclosed or submitted for publication or presentation without the Sponsor’s prior written agreement.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Milica Enoiu / Clinical Research Manager
Organization: Vifor Fresenius Medical Care Renal Pharma France
Phone: +41588518264
EMail: milica.enoiu@viforpharma.com
Layout table for additonal information
Responsible Party: Vifor Fresenius Medical Care Renal Pharma
ClinicalTrials.gov Identifier: NCT02688764    
Other Study ID Numbers: PA-CL-PED-01
First Submitted: February 17, 2016
First Posted: February 23, 2016
Results First Submitted: August 8, 2019
Results First Posted: August 28, 2019
Last Update Posted: September 10, 2019