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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

Tracking Information
First Submitted Date  ICMJE February 17, 2016
First Posted Date  ICMJE February 23, 2016
Results First Submitted Date  ICMJE August 8, 2019
Results First Posted Date  ICMJE August 28, 2019
Last Update Posted Date September 10, 2019
Actual Study Start Date  ICMJE May 26, 2016
Actual Primary Completion Date February 21, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 28, 2019)
  • Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the PA21 Group [ Time Frame: From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date) ]
  • Number and Percentage of Participants Who Withdrew Due to Treatment Emergent Adverse Events [ Time Frame: through study completion, up to 34 weeks after treatment start date ]
    Any adverse event Leading to Study Drug Withdrawal is considered.
  • Number and Percentage of Participants With Any Treatment Emergent Adverse Event [ Time Frame: through study completion, up to 34 weeks after treatment start date ]
    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.
Original Primary Outcome Measures  ICMJE
 (submitted: February 22, 2016)
Change in serum phosphorus from baseline in the PA21 group [ Time Frame: At the end of Stage 1 (up to 10 weeks after randomization) ]
Change History Complete list of historical versions of study NCT02688764 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: August 28, 2019)
  • Change in Serum Phosphorus Level From Baseline to the End of Stage 1 in the Phoslyra Group [ Time Frame: From Baseline to the End of Stage 1 (up to 10 weeks after treatment start date) ]
  • Change in Serum Phosphorus Level From Baseline to the End of Stage 2 in Both Groups [ Time Frame: From baseline to study completion, up to 34 weeks after treatment start date ]
  • Participants With Serum Phosphorus Levels Within the Age-dependent Target Range in Each Stage [ Time Frame: through study completion, up to 34 weeks after treatment start date ]
    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
  • Participants With Serum Phosphorus Levels Within the Age Related Normal Range in Each Stage [ Time Frame: through study completion, up to 34 weeks after treatment start date ]
    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
  • Serum Phosphorus Values at Each Visit [ Time Frame: through study completion, up to 34 weeks after treatment start date ]
  • Serum Total Corrected Calcium at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Participants With Sustained Hypercalcaemia [ Time Frame: through study completion, up to 34 weeks after treatment start date ]
    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
  • Serum Total Corrected Calcium-Phosphorus Product at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
    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
  • Serum iPTH Levels at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Ferritin Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Unsaturated Iron Binding Capacity Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Iron Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Transferrin Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • 25-Hydroxy Vitamin D Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Bone Specific Alkaline Phosphatase Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Type I Collagen C-Telopeptides Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Fibroblast Growth Factor 23 Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Osteocalcin-CL Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
  • Tartrate-resistant Acid Phosphatase 5b Values at Each Time Point and Change From Baseline [ Time Frame: From baseline through study completion, up to 34 weeks after treatment start date ]
Original Secondary Outcome Measures  ICMJE
 (submitted: February 22, 2016)
  • Change in serum phosphorus from baseline in the Phoslyra group [ Time Frame: At end of Stage 1 (up to 10 weeks after randomization) ]
  • Change in serum phosphorus from baseline in the PA21 group [ Time Frame: through study completion, an average of 34 weeks ]
  • Change in serum phosphorus from baseline in the Phoslyra group [ Time Frame: through study completion, an average of 34 weeks ]
  • Percentage of subjects in each stage during which the subject has serum phosphorus levels in the age dependent target ranges. [ Time Frame: through study completion, an average of 34 weeks ]
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE 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
Official Title  ICMJE An Open-label, Randomised, Active-controlled, Parallel Group, Multicentre, Phase 3 Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent CKD Patients With Hyperphosphataemia
Brief Summary This is a Phase 3, Open-label, Randomised, Active-controlled, Parallel Group, Multicentre Study to Investigate the Safety and Efficacy of PA21 (Velphoro®) and Calcium Acetate (Phoslyra®) in Paediatric and Adolescent CKD Patients with Hyperphosphataemia. The aim of this Phase 3 clinical study is to demonstrate similar efficacy of PA21 (Velphoro) in paediatric and adolescent patients with CKD, and to provide safety and dosing information for this patient population. The Phoslyra (comparator) group provides information for a descriptive comparison of PA21 against a commonly used calcium-based phosphate binder (calcium acetate).
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 3
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Hyperphosphatemia
Intervention  ICMJE
  • Drug: PA21 (Velphoro®)

    During Stage 1 (Open-Label Dose Titration; up to 10 weeks), PA21 subjects will receive PA21 at a starting dose based on their age. Dose of PA21 will be increased or decreased as required for efficacy (to achieve age specific target serum phosphorus level), provided a subject has been receiving that dose for a minimum of 2 weeks, and for safety or tolerability reasons at any time.

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

    Other Name: sucroferric oxyhydroxide
  • Drug: Calcium Acetate (Phoslyra®)

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

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

Study Arms  ICMJE
  • Experimental: PA21 (Velphoro®)

    PA21 (Velphoro®), chewable tablets 500 mg iron

    PA21 (Velphoro®), chewable tablets 250 mg iron

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

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

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

    Intervention: Drug: PA21 (Velphoro®)
  • Active Comparator: Calcium Acetate (Phoslyra®)
    Calcium Acetate (Phoslyra®) - Oral Solution: 667 mg calcium acetate per 5 mL.
    Intervention: Drug: Calcium Acetate (Phoslyra®)
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Terminated
Actual Enrollment  ICMJE
 (submitted: April 17, 2019)
85
Original Estimated Enrollment  ICMJE
 (submitted: February 22, 2016)
130
Actual Study Completion Date  ICMJE February 21, 2019
Actual Primary Completion Date February 21, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Inclusion Criteria:

  1. Subjects 0 to <18 years at time of consent.
  2. Subjects with hyperphosphataemia
  3. Subjects ≥1 year with CKD Stages 4-5 defined by a glomerular filtration rate <30 mL/min/1.73 m2 or with CKD Stage 5D receiving adequate maintenance haemodialysis (HD) or peritoneal dialysis (PD) for at least 2 months prior to screening.
  4. Subjects <1 year must have CKD.
  5. Appropriate written informed consent and, where appropriate/required assent, have been provided.

Exclusion Criteria:

  1. Subjects with hypercalcaemia at screening
  2. Subjects with intact parathyroid hormone (iPTH) levels >700 pg/mL at screening.
  3. Subjects who are PB naïve who weigh <5 kg at screening. Subjects receiving stable doses of PBs who weigh <6 kg at screening
  4. Subjects requiring feeding tube sizes ≤6 FR (French catheter scale).
  5. Subjects with history of major gastrointestinal surgery or significant gastrointestinal disorders.
  6. Subjects with hypocalcaemia (serum total corrected calcium <1.9 mmol/L; <7.6 mg/dL) at screening.
  7. Subject is pregnant (e.g., positive human chorionic gonadotropin test) or breast feeding.
  8. Subject has a significant medical condition(s)
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE up to 18 Years   (Child, Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE France,   Germany,   Lithuania,   Poland,   Romania,   Russian Federation,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02688764
Other Study ID Numbers  ICMJE PA-CL-PED-01
Has Data Monitoring Committee Yes
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Vifor Fresenius Medical Care Renal Pharma
Study Sponsor  ICMJE Vifor Fresenius Medical Care Renal Pharma
Collaborators  ICMJE Not Provided
Investigators  ICMJE
Principal Investigator: Larry A Greenbaum, MD; PhD Children's Healthcare of Atlanta at Egleston
PRS Account Vifor Fresenius Medical Care Renal Pharma
Verification Date August 2019

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP