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Study to Evaluate the Safety and Efficacy of Phenoptin™ in Subjects With Phenylketonuria Who Have Elevated Phenylalanine Levels

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ClinicalTrials.gov Identifier: NCT00104247
Recruitment Status : Completed
First Posted : February 25, 2005
Results First Posted : April 16, 2009
Last Update Posted : July 23, 2014
Sponsor:
Information provided by:
BioMarin Pharmaceutical

Study Type Interventional
Study Design Allocation: Randomized;   Intervention Model: Parallel Assignment;   Masking: Double;   Primary Purpose: Treatment
Condition Phenylketonurias
Intervention Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin
Enrollment 89
Recruitment Details  
Pre-assignment Details  
Arm/Group Title Sapropterin Dihydrochloride Placebo
Hide Arm/Group Description Patients administered 10 mg /kg orally once daily. Placebo
Period Title: Overall Study
Started 42 47
Completed 41 [1] 46 [2]
Not Completed 1 1
Reason Not Completed
Withdrawal by Subject             1             0
Noncompliant with dosing             0             1
[1]
Subject withdrew prior to dosing
[2]
One subject withdrew at Week 4, but is included in the data analysis
Arm/Group Title Sapropterin Dihydrochloride Placebo Total
Hide Arm/Group Description Patients administered 10 mg /kg orally once daily. Placebo Total of all reporting groups
Overall Number of Baseline Participants 41 47 88
Hide Baseline Analysis Population Description
[Not Specified]
Age, Continuous  
Mean (Standard Deviation)
Unit of measure:  Years
Number Analyzed 41 participants 47 participants 88 participants
21.5  (9.5) 19.5  (9.8) 20.4  (9.7)
Age, Customized  
Measure Type: Number
Unit of measure:  Years
Number Analyzed 41 participants 47 participants 88 participants
8 <= Age <= 12 6 11 17
12 < Age 35 36 71
Sex: Female, Male  
Measure Type: Count of Participants
Unit of measure:  Participants
Number Analyzed 41 participants 47 participants 88 participants
Female
14
  34.1%
23
  48.9%
37
  42.0%
Male
27
  65.9%
24
  51.1%
51
  58.0%
Race/Ethnicity, Customized  
Measure Type: Number
Unit of measure:  Participants
Number Analyzed 41 participants 47 participants 88 participants
Asian/Pacific Islander 1 0 1
Caucasian 39 47 86
Other 1 0 1
1.Primary Outcome
Title Change in Blood Phenylalanine Levels From Baseline to Week 6.
Hide Description [Not Specified]
Time Frame baseline to week 6
Hide Outcome Measure Data
Hide Analysis Population Description
[Not Specified]
Arm/Group Title Sapropterin Dihydrochloride Placebo
Hide Arm/Group Description:
Patients administered 10 mg /kg orally once daily.
Placebo
Overall Number of Participants Analyzed 41 46
Mean (Standard Deviation)
Unit of Measure: micromole per liter
Baseline Blood Phe Level 843  (300) 888  (323)
Week 6 Blood Phe Level 607  (377) 891  (348)
Mean Change in Blood Phe from Baseline to Week 6 -239  (38) 6  (36)
Show Statistical Analysis 1 Hide Statistical Analysis 1
Statistical Analysis Overview Comparison Group Selection Sapropterin Dihydrochloride, Placebo
Comments [Not Specified]
Type of Statistical Test Superiority or Other
Comments [Not Specified]
Statistical Test of Hypothesis P-Value <0.001
Comments [Not Specified]
Method ANCOVA
Comments [Not Specified]
Time Frame [Not Specified]
Adverse Event Reporting Description [Not Specified]
 
Arm/Group Title Sapropterin Dihydrochloride Placebo
Hide Arm/Group Description Patients administered 10 mg /kg orally once daily. Placebo
All-Cause Mortality
Sapropterin Dihydrochloride Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   --/--   --/-- 
Show Serious Adverse Events Hide Serious Adverse Events
Sapropterin Dihydrochloride Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   0/41 (0.00%)   0/47 (0.00%) 
Show Other (Not Including Serious) Adverse Events Hide Other (Not Including Serious) Adverse Events
Frequency Threshold for Reporting Other Adverse Events 5%
Sapropterin Dihydrochloride Placebo
Affected / at Risk (%) Affected / at Risk (%)
Total   21   34 
Gastrointestinal disorders     
Vomiting   2/41 (4.88%)  4/47 (8.51%) 
Abdominal Pain   1/41 (2.44%)  4/47 (8.51%) 
Diarrhoea   2/41 (4.88%)  3/47 (6.38%) 
General disorders     
Pyrexia   2/41 (4.88%)  2/47 (4.26%) 
Infections and infestations     
Upper Respiratory Tract Infection   7/41 (17.07%)  13/47 (27.66%) 
Pharyngitis   2/41 (4.88%)  1/47 (2.13%) 
Infection   2/41 (4.88%)  0/47 (0.00%) 
Musculoskeletal and connective tissue disorders     
Back Pain   1/41 (2.44%)  3/47 (6.38%) 
Nervous system disorders     
Headache   4/41 (9.76%)  7/47 (14.89%) 
Respiratory, thoracic and mediastinal disorders     
Cough   0/41 (0.00%)  3/47 (6.38%) 
Pharynholaryngeal Pain   3/41 (7.32%)  0/47 (0.00%) 
Indicates events were collected by systematic assessment
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.
The first publication of the results shall be made by Sponsor in a joint publication. If such a multi-center publication is not submitted within 12 months after conclusion of the study, the PI may publish the results from their site individually, subject however to comply with the other terms of the agreement.
Results Point of Contact
Layout table for Results Point of Contact information
Name/Title: Medical Information Services
Organization: BioMarin Pharmaceutical Inc.
Phone: 1-800-983-4587
EMail: medinfo@bmrn.com
Layout table for additonal information
ClinicalTrials.gov Identifier: NCT00104247     History of Changes
Other Study ID Numbers: PKU-003
First Submitted: February 24, 2005
First Posted: February 25, 2005
Results First Submitted: January 29, 2009
Results First Posted: April 16, 2009
Last Update Posted: July 23, 2014