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Trial record 7 of 9 for:    "Hereditary Hypophosphatemic Rickets" | "Calcium"

Effectiveness of Paricalcitol in Reducing Parathyroid Hormone (PTH) Levels in X-linked Hypophosphatemic Rickets

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ClinicalTrials.gov Identifier: NCT00417612
Recruitment Status : Completed
First Posted : January 1, 2007
Results First Posted : October 28, 2014
Last Update Posted : October 28, 2014
Sponsor:
Collaborator:
National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)
Information provided by (Responsible Party):
Thomas Carpenter, Yale University

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor);   Primary Purpose: Treatment
Conditions Hypophosphatemia, Familial
Hyperparathyroidism
Interventions Drug: Paricalcitol
Other: Placebo
Enrollment 33
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description

Participants given active drug, paricalcitol (Zemplar), in effort to reduce Parathyroid Hormone (PTH) level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Period Title: Overall Study
Started 22 11
Completed 19 8
Not Completed 3 3
Reason Not Completed
Errant diagnosis             0             1
Adverse Event             2             0
Protocol Violation             0             2
Lost to Follow-up             1             0
Arm/Group Title Paricalcitol (Active Drug) Placebo Total
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Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Total of all reporting groups
Overall Number of Baseline Participants 22 11 33
Hide Baseline Analysis Population Description
[Not Specified]
Age, Categorical  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants 11 participants 33 participants
<=18 years
2
   9.1%
1
   9.1%
3
   9.1%
Between 18 and 65 years
19
  86.4%
10
  90.9%
29
  87.9%
>=65 years
1
   4.5%
0
   0.0%
1
   3.0%
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 22 participants 11 participants 33 participants
Female
17
  77.3%
9
  81.8%
26
  78.8%
Male
5
  22.7%
2
  18.2%
7
  21.2%
Region of Enrollment  
Measure Type: Number
Unit of measure:  Participants
United States Number Analyzed 22 participants 11 participants 33 participants
22 11 33
1.Primary Outcome
Title Area Under the Curve for Parathyroid Hormone (PTHauc) Measurement
Hide Description Mean area under the curve for parathyroid hormone levels (PTHauc) sampled during a 26 hour study period, for Paricalcitol (Active Drug) and Placebo groups at Baseline and Month 12 .
Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Overall Number of Participants Analyzed 19 8
Mean (Standard Deviation)
Unit of Measure: nlEq*26hr/ml
Baseline 1223  (426) 1449  (865)
Month 12 987  (372) 1616  (945)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Paricalcitol (Active Drug), Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value 0.007
Comments [Not Specified]
Method ANOVA
Comments [Not Specified]
2.Primary Outcome
Title Area Under the Curve for Parathyroid Hormone (PTH; Percentage Decrease)
Hide Description Mean PTHauc (% decrease) for Paricalcitol (Active Drug) and Placebo from Baseline to Month 12.
Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce parathyroid hormone (PTH) level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Overall Number of Participants Analyzed 19 8
Measure Type: Number
Unit of Measure: participants
20-29% 5 1
30-39% 1 1
>/= 40% 3 0
3.Primary Outcome
Title Reduction of Parathyroid Hormone Area Under the Curve (PTHauc) of 20% or Greater
Hide Description Clinically significant reduction of Mean PTHauc (% decrease >/= 20) for Paricalcitol (Active Drug) and Placebo from Baseline to Month 12.
Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Overall Number of Participants Analyzed 19 8
Measure Type: Number
Unit of Measure: percentage of participants
53 25
4.Secondary Outcome
Title Static Parameters of Serum Alkaline Phosphatase at Baseline and 1 Year for Paricalcitol and Placebo Arms.
Hide Description [Not Specified]
Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
Alkaline phosphatase activity was analyzed separately for children (less than 18 years of age) and adults as values varied considerably between these two groups. In other outcome measure modules the paricalcitol group is comprised of 19 participants analyzed which includes adults and children. In this module, the children (2) are their own arm.
Arm/Group Title Paricalcitol (Active Drug) Placebo Paricalcitol (Children Ages 9-17)
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Pediatric patients ages 9-17 given paricalcitol
Overall Number of Participants Analyzed 17 8 2
Mean (Standard Deviation)
Unit of Measure: mg/dl
Serum Alkaline Phosphatase (Baseline) 141  (96) 96  (28) 354  (2)
Serum Alkaline Phosphatase (1 Year) 112  (70) 98  (34) 402  (211)
5.Secondary Outcome
Title Serum Calcium
Hide Description [Not Specified]
Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Overall Number of Participants Analyzed 19 8
Mean (Standard Deviation)
Unit of Measure: mg/dl
Serum Calcium (Baseline) 9.2  (0.5) 9.3  (0.5)
Serum Calcium (1 Year) 9.4  (0.5) 9.3  (0.5)
6.Secondary Outcome
Title Bone Scan Severity Score
Hide Description

99-Tc-methylenediphosphonate bone scans were completed and analyzed using the following scale*: Bone scan severity scale with minimum score of 0 and maximum score of 4 (0 is no disease and 4 is greatest severity of disease).

Appearance of lumbar spine and the sacroiliac region were used as internal references to which all suspected lesions were compared, and scored as follows:

grade 0, normal scan without suspicious lesions grade 1, lesion(s) less intense than normal lumbar spine grade 2, lesion(s) similar in intensity to normal lumbar spine grade 3, lesions more intense than normal lumbar spine but similar to the normal sacroiliac region grade 4, lesions more intense than the normal sacroiliac region.

*devised by nuclear medicine radiologist at Yale New Haven Hospital

Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Overall Number of Participants Analyzed 19 8
Mean (Standard Deviation)
Unit of Measure: units on a scale
Baseline Scan Score 2.18  (1.19) 2.00  (1.51)
12 Month Scan Score 1.76  (1.25) 2.12  (1.55)
7.Secondary Outcome
Title Percent Change in Urinary Calcium Excretion From Baseline to 1 Year
Hide Description Percent change in daily urinary calcium excretion, which is calculated the measurements of the calcium in the subject's 24-hr urine collections done at baseline and at the 1 year timepoints
Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Overall Number of Participants Analyzed 19 8
Mean (Standard Deviation)
Unit of Measure: percent change
253  (388) 9  (36)
8.Secondary Outcome
Title Serum Intact Fibroblast Growth Factor 23 (FGF23)
Hide Description [Not Specified]
Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Overall Number of Participants Analyzed 19 8
Mean (95% Confidence Interval)
Unit of Measure: pg/ml
Serum Intact FGF23 (Baseline)
120
(72 to 203)
93
(46 to 188)
Serum Intact FGF23 (1 Year)
244
(145 to 411)
104
(47 to 227)
9.Secondary Outcome
Title Serum 1,25 (OH)2D
Hide Description [Not Specified]
Time Frame Measured at baseline and Month 12
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description:

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

Overall Number of Participants Analyzed 19 8
Mean (Standard Deviation)
Unit of Measure: pg/ml
Serum 1,25(OH)2D (Baseline) 51  (18) 47  (9)
Serum 1,25 (OH)2D (1 Year) 52  (17) 46  (14)
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Paricalcitol (Active Drug) Placebo
Hide Arm/Group Description

Participants given active drug, paricalcitol (Zemplar), in effort to reduce PTH level

Paricalcitol: Paricalcitol given first as a dose of 2 capsules once per day. Dose titration as needed per biochemical results at outpatient visits.

Participants given placebo capsule to match for comparison

Placebo: Placebo sugar pill

All-Cause Mortality
Paricalcitol (Active Drug) Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--      --/--    
Show Serious Adverse Events Hide Serious Adverse Events
Paricalcitol (Active Drug) Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   0/22 (0.00%)      0/11 (0.00%)    
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 0%
Paricalcitol (Active Drug) Placebo
Affected / at Risk (%) # Events Affected / at Risk (%) # Events
Total   2/22 (9.09%)      0/11 (0.00%)    
Blood and lymphatic system disorders     
Hypercalcemia  1/22 (4.55%)  1 0/11 (0.00%)  0
Renal and urinary disorders     
Elevated Serum Creatinine  1/22 (4.55%)  1 0/11 (0.00%)  0
Certain Agreements
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT 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.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Dr. Thomas O. Carpenter
Organization: Yale University School of Medicine
Phone: 203-785-6526
EMail: thomas.carpenter@yale.edu
Layout table for additonal information
Responsible Party: Thomas Carpenter, Yale University
ClinicalTrials.gov Identifier: NCT00417612     History of Changes
Other Study ID Numbers: P50AR054086 ( U.S. NIH Grant/Contract )
P50AR054086 ( U.S. NIH Grant/Contract )
1P50AR054086-01 ( U.S. NIH Grant/Contract )
First Submitted: December 28, 2006
First Posted: January 1, 2007
Results First Submitted: August 2, 2014
Results First Posted: October 28, 2014
Last Update Posted: October 28, 2014