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History of Changes for Study: NCT00943579
Open-Label Extension Study of Kuvan for Autism
Latest version (submitted April 30, 2018) on ClinicalTrials.gov
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Study Record Versions
Version A B Submitted Date Changes
1 July 20, 2009 None (earliest Version on record)
2 August 14, 2009 Recruitment Status, Study Status, Contacts/Locations and Oversight
3 January 15, 2010 Study Status and Study Description
4 May 25, 2010 Study Status
5 May 11, 2011 Study Status
6 December 22, 2011 Sponsor/Collaborators, Study Status and Study Design
7 August 31, 2012 Recruitment Status and Study Status
8 July 2, 2013 Study Status, Outcome Measures, Results, Eligibility, Study Design and Study Description
9 January 23, 2018 Study Status, Outcome Measures and Baseline Characteristics
10 April 30, 2018 Outcome Measures, Study Status and Study Description
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Study NCT00943579
Submitted Date:  July 20, 2009 (v1)

Open or close this module Study Identification
Unique Protocol ID: CHC-0902
Brief Title: Open-Label Extension Study of Kuvan for Autism
Official Title: Kuvan® (Sapropterin) as a Treatment for Autistic Disorder: An Open Label Extension Protocol
Secondary IDs:
Open or close this module Study Status
Record Verification: July 2009
Overall Status: Not yet recruiting
Study Start: August 2009
Primary Completion: June 2010 [Anticipated]
Study Completion: June 2010 [Anticipated]
First Submitted: July 20, 2009
First Submitted that
Met QC Criteria:
July 20, 2009
First Posted: July 22, 2009 [Estimate]
Last Update Submitted that
Met QC Criteria:
July 20, 2009
Last Update Posted: July 22, 2009 [Estimate]
Open or close this module Sponsor/Collaborators
Sponsor: The Children's Health Council
Responsible Party:
Collaborators: BioMarin Pharmaceutical
Open or close this module Oversight
U.S. FDA-regulated Drug:
U.S. FDA-regulated Device:
Data Monitoring: No
Open or close this module Study Description
Brief Summary: 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. The purpose of the study primarily is to gather additional information on safety and efficacy in this population.
Detailed Description:
Open or close this module Conditions
Conditions: Autistic Disorder
Keywords: autism
autistic disorder
tetrahydrobiopterin
sapropterin
treatment
Open or close this module Study Design
Study Type: Interventional
Primary Purpose: Treatment
Study Phase: Phase 2/Phase 3
Interventional Study Model: Single Group Assignment
Number of Arms: 1
Masking: None (Open Label)
Allocation: N/A
Enrollment: 56 [Anticipated]
Open or close this module Arms and Interventions
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:
  • sapropterin
  • tetrahydrobiopterin
Open or close this module Outcome Measures
Primary Outcome Measures:
1. Clinical Global Impressions Scale
[ Time Frame: 16 weeks ]

Secondary Outcome Measures:
1. Vineland Adaptive Behavior Scale
[ Time Frame: Weeks 8 and 16 ]

2. Clinical Global Impression: Severity
[ Time Frame: Weeks 8 & 16 ]

3. Children's Yale Brown Obsessive Compulsive Scale
[ Time Frame: Weeks 8 & 16 ]

4. Parental Global Assessment
[ Time Frame: Weeks 8 & 16 ]

5. Preschool Language Scale
[ Time Frame: Weeks 8 & 16 ]

6. Connor's Preschool ADHD questionnaire
[ Time Frame: Weeks 8 & 16 ]

7. Aberrant Behavior Checklist
[ Time Frame: Weeks 8 & !6 ]

8. Adverse Events Reporting
[ Time Frame: Cummulative throughout study ]

Open or close this module Eligibility
Minimum Age: 3 Years
Maximum Age: 6 Years
Sex: All
Gender Based:
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
Open or close this module Contacts/Locations
Central Contact Person: Glen R Elliott, PhD, MD
Telephone: 6506883649
Email: gelliott@chconline.org
Central Contact Backup: Lynne Huffman, MD
Telephone: 6506883609
Email: lhuffman@chconline.org
Study Officials: Glen R Elliott, PhD, MD
Principal Investigator
The Children's Health Council
Locations: United States, California
The Children's Health Council
Palo Alto, California, United States, 94304
Open or close this module IPDSharing
Plan to Share IPD:
Open or close this module References
Citations:
Links:
Available IPD/Information:

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