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

This study has been completed.
Sponsor:
Information provided by:
BioMarin Pharmaceutical
ClinicalTrials.gov Identifier:
NCT00104247
First received: February 24, 2005
Last updated: September 24, 2009
Last verified: September 2009
Results First Received: January 29, 2009  
Study Type: Interventional
Study Design: Allocation: Randomized;   Endpoint Classification: Safety/Efficacy Study;   Intervention Model: Parallel Assignment;   Masking: Double-Blind;   Primary Purpose: Treatment
Condition: Phenylketonurias
Intervention: Drug: sapropterin dihydrochloride, 6R-BH4, tetrahydrobiopterin

  Participant Flow
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Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
No text entered.

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Sapropterin Dihydrochloride Patients administered 10 mg /kg orally once daily.
Placebo Placebo

Participant Flow:   Overall Study
    Sapropterin Dihydrochloride     Placebo  
STARTED     42     47  
COMPLETED     41 [1]   46 [2]
NOT COMPLETED     1     1  
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



  Baseline Characteristics
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Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Sapropterin Dihydrochloride Patients administered 10 mg /kg orally once daily.
Placebo Placebo
Total Total of all reporting groups

Baseline Measures
    Sapropterin Dihydrochloride     Placebo     Total  
Number of Participants  
[units: participants]
  41     47     88  
Age  
[units: years]
Mean ± Standard Deviation
  21.5  ± 9.5     19.5  ± 9.8     20.4  ± 9.7  
Age, Customized  
[units: Years]
     
8 <= Age <= 12     6     11     17  
12 < Age     35     36     71  
Gender  
[units: participants]
     
Female     14     23     37  
Male     27     24     51  
Race/Ethnicity, Customized  
[units: participants]
     
Asian/Pacific Islander     1     0     1  
Caucasian     39     47     86  
Other     1     0     1  



  Outcome Measures

1.  Primary:   Change in Blood Phenylalanine Levels From Baseline to Week 6.   [ Time Frame: baseline to week 6 ]


  Serious Adverse Events


  Other Adverse Events


  More Information
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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 agreement is:
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is less than or equal to 60 days. The sponsor cannot require changes to the communication and cannot extend the embargo.
unchecked The only disclosure restriction on the PI is that the sponsor can review results communications prior to public release and can embargo communications regarding trial results for a period that is more than 60 days but less than or equal to 180 days. The sponsor cannot require changes to the communication and cannot extend the embargo.


Limitations and Caveats
Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
Not Applicable  


Results Point of Contact:  
Name/Title: Medical Information Services
Organization: BioMarin Pharmaceutical Inc.
phone: 1-800-983-4587
e-mail: medinfo@bmrn.com


No publications provided by BioMarin Pharmaceutical

Publications automatically indexed to this study:

ClinicalTrials.gov Identifier: NCT00104247     History of Changes
Other Study ID Numbers: PKU-003
Study First Received: February 24, 2005
Results First Received: January 29, 2009
Last Updated: September 24, 2009
Health Authority: United States: Food and Drug Administration