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Tetrahydrobiopterin Treatment in Children With Idiopathic Cognitive Developmental Disorders

This study has been completed.
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Richard Frye, Arkansas Children's Hospital Research Institute
ClinicalTrials.gov Identifier:
NCT01141595
First received: June 8, 2010
Last updated: February 28, 2014
Last verified: February 2014
Results First Received: January 15, 2013  
Study Type: Interventional
Study Design: Intervention Model: Single Group Assignment;   Masking: Open Label;   Primary Purpose: Treatment
Condition: Autism Spectrum Disorder
Intervention: Drug: sapropterin dihydrochloride

  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
All patients were recruited from a medical clinic specalizing in neurodevelopmental disorders

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

Reporting Groups
  Description
Kuvan® Patients will be instructed to take 20 mg/kg/day of Kuvan® orally dissolved in 4 - 8oz. of water or apple juice with breakfast.

Participant Flow:   Overall Study
    Kuvan®  
STARTED     10  
COMPLETED     8  
NOT COMPLETED     2  



  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
Kuvan® Patients will be instructed to take 20 mg/kg/day of Kuvan® orally dissolved in 4 - 8oz. of water or apple juice with breakfast.

Baseline Measures
    Kuvan®  
Number of Participants  
[units: participants]
  10  
Age  
[units: participants]
 
<=18 years     10  
Between 18 and 65 years     0  
>=65 years     0  
Age  
[units: years]
Mean ± Standard Deviation
  5.0  ± 1.2  
Gender  
[units: participants]
 
Female     1  
Male     9  
Region of Enrollment  
[units: participants]
 
United States     10  



  Outcome Measures

1.  Primary:   Preschool Language Scales   [ Time Frame: 16 weeks ]


  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
No text entered.


  More Information
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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:  
Name/Title: Richard Frye
Organization: Arkansas Children's Hospital Reseach Insitute
phone: 501-364-5465
e-mail: REFrye@uams.edu


Publications of Results:
Other Publications:

Responsible Party: Richard Frye, Arkansas Children's Hospital Research Institute
ClinicalTrials.gov Identifier: NCT01141595     History of Changes
Other Study ID Numbers: HSC-MS-09-0578
Study First Received: June 8, 2010
Results First Received: January 15, 2013
Last Updated: February 28, 2014
Health Authority: United States: Institutional Review Board