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Open-Label Extension Study of Kuvan for Autism

This study has been completed.
Sponsor:
Collaborator:
BioMarin Pharmaceutical
Information provided by (Responsible Party):
Glen R. Elliott, The Children's Health Council
ClinicalTrials.gov Identifier:
NCT00943579
First received: July 20, 2009
Last updated: August 31, 2012
Last verified: August 2012
History of Changes
  Purpose

This is an open-label extension study available only to subjects who completed an earlier double-blind, placebo-controlled study of sapropterin in children with autism. During this protocol, all subjects will be receiving brand-name Kuvan 20 mg/kg/day for 16 weeks; subject who complete the first 16 weeks will have the option of continuing on Kuvan at the same dose for up to 90 days after the last subject has completed the first 16 weeks of this protocol. The purpose of the study primarily is to gather additional information on safety and efficacy in this population.


Condition Intervention Phase
Autistic Disorder
 Drug: Kuvan®
 Phase 2
Phase 3

Study Type: Interventional
Study Design: Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Kuvan® (Sapropterin) as a Treatment for Autistic Disorder: An Open Label Extension Protocol

Resource links provided by NLM:

MedlinePlus related topics: Autism
Drug Information available for: Sapropterin
U.S. FDA Resources

Further study details as provided by The Children's Health Council:

Primary Outcome Measures:
  • Clinical Global Impressions Scale [ Time Frame: 16 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Vineland Adaptive Behavior Scale [ Time Frame: Weeks 8 and 16 ] [ Designated as safety issue: No ]
  • Clinical Global Impression: Severity [ Time Frame: Weeks 8 & 16 ] [ Designated as safety issue: No ]
  • Children's Yale Brown Obsessive Compulsive Scale [ Time Frame: Weeks 8 & 16 ] [ Designated as safety issue: No ]
  • Parental Global Assessment [ Time Frame: Weeks 8 & 16 ] [ Designated as safety issue: No ]
  • Preschool Language Scale [ Time Frame: Weeks 8 & 16 ] [ Designated as safety issue: No ]
  • Connor's Preschool ADHD questionnaire [ Time Frame: Weeks 8 & 16 ] [ Designated as safety issue: No ]
  • Aberrant Behavior Checklist [ Time Frame: Weeks 8 & !6 ] [ Designated as safety issue: No ]
  • Adverse Events Reporting [ Time Frame: Cummulative throughout study ] [ Designated as safety issue: Yes ]

Enrollment: 35
Study Start Date: August 2009
Study Completion Date: March 2012
Primary Completion Date: December 2011 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Kuvan®
Kuvan® (sapropterin) will be administered to all subjects at 20 mg/kg/day for 16 weeks.
 Drug: Kuvan®
Brand-name Kuvan® (sapropterin) will be administered to all subjects at a dose of 20 mg/kg/day for 16 weeks.
Other Names:
  • Kuvan®
  • sapropterin
  • tetrahydrobiopterin

  Eligibility

Ages Eligible for Study:   3 Years to 6 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • All subjects must have completed earlier trial, CHC 0901
  • Parents must be willing and able to sign informed consent

Exclusion Criteria:

  • Child failed to complete CHC 0901
  Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00943579

Locations
United States, California
The Children's Health Council
Palo Alto, California, United States, 94304
Sponsors and Collaborators
The Children's Health Council
BioMarin Pharmaceutical
Investigators
Principal Investigator: Glen R Elliott, PhD, MD The Children's Health Council
  More Information

No publications provided

Responsible Party: Glen R. Elliott, Chief Psychiatrist and Medical Director, The Children's Health Council
ClinicalTrials.gov Identifier: NCT00943579     History of Changes
Other Study ID Numbers: CHC-0902
Study First Received: July 20, 2009
Last Updated: August 31, 2012
Health Authority: United States: Institutional Review Board

Keywords provided by The Children's Health Council:
autism
autistic disorder
tetrahydrobiopterin
sapropterin
treatment

Additional relevant MeSH terms:
Autistic Disorder
Child Development Disorders, Pervasive
Mental Disorders Diagnosed in Childhood
Mental Disorders

ClinicalTrials.gov processed this record on May 23, 2013