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

This study has been completed.
Sponsor:
ClinicalTrials.gov Identifier:
NCT00104247
First Posted: February 25, 2005
Last Update Posted: July 23, 2014
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.
Information provided by:
BioMarin Pharmaceutical
Results First Submitted: January 29, 2009  
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

  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 
Overall Participants Analyzed 
[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


  Limitations and Caveats
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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
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  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.


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


Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):

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