Sapropterin as a Treatment for Autistic Disorder
The recruitment status of this study is unknown because the information has not been verified recently.
Verified May 2011 by The Children's Health Council.
Recruitment status was Active, not recruiting
Recruitment status was Active, not recruiting
Sponsor:
The Children's Health Council
Collaborator:
BioMarin Pharmaceutical
Information provided by:
The Children's Health Council
ClinicalTrials.gov Identifier:
NCT00850070
First received: February 20, 2009
Last updated: May 11, 2011
Last verified: May 2011
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Purpose
This study is intended to provide a definitive test of the hypothesis that elevating sapropterin (tetrahydrobiopterin, a cofactor for several key brain enzymes)concentrations in the CNS will result in measurable improvements in core symptoms of autism in young individuals, under age 6 years. The study will entail a double-blind, placebo-controlled 16-week intervention.
| Condition | Intervention | Phase |
|---|---|---|
|
Autistic Disorder |
Drug: sapropterin Drug: Placebo |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Allocation: Randomized Endpoint Classification: Safety/Efficacy Study Intervention Model: Parallel Assignment Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor) Primary Purpose: Treatment |
| Official Title: | Sapropterin as a Treatment for Autistic Disorder: A Phase II Randomized, Double-Blind, Placebo-Controlled Trial |
Resource links provided by NLM:
Further study details as provided by The Children's Health Council:
Primary Outcome Measures:
- Clinical Global Impression -- Improvement (CGI-I) Scale [ Time Frame: Weekly for 4 weeks, then monthly, with 16-week end point ] [ Designated as safety issue: No ]
Secondary Outcome Measures:
- Preschool Language Scale (PLS) [ Time Frame: Baseline, 8 weeks, and 16 weeks ] [ Designated as safety issue: No ]
- Vineland Adaptive Behavior Scale-II [ Time Frame: Baseline, 8 weeks, and 16 weeks ] [ Designated as safety issue: No ]
- Children's Yale Brown Obsessive Compulsive Scale (C-YBOCS) [ Time Frame: Baseline, 8 weeks, and 16 weeks ] [ Designated as safety issue: No ]
- Connor's Preschool ADHD questionnaire [ Time Frame: Baseline, 8 weeks, and 16 weeks ] [ Designated as safety issue: No ]
- Adverse Events Scale [ Time Frame: Every 1-2 weeks for 16 weeks ] [ Designated as safety issue: Yes ]
- Aberrant Behavior Checklist (ABC) - Irritability Subscale [ Time Frame: Baseline, 8 weeks, and 16 weeks ] [ Designated as safety issue: No ]
- Clinical Global Impression -- Severity (CGI-S) Scale [ Time Frame: Baseline, 8 weeks, and 16 weeks ] [ Designated as safety issue: No ]
- Social Responsiveness Scale (SRS) [ Time Frame: Baseline, 8 weeks, and 16 weeks ] [ Designated as safety issue: No ]
- Parent Global Assessment (PGA) Scale [ Time Frame: Baseline, 8 weeks, and 16 weeks ] [ Designated as safety issue: No ]
| Estimated Enrollment: | 56 |
| Study Start Date: | March 2009 |
| Estimated Study Completion Date: | August 2011 |
| Estimated Primary Completion Date: | May 2011 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| Experimental: sapropterin |
Drug: sapropterin
Patients will receive sapropterin 20 mg per kilogram per day for 16 weeks
Other Names:
|
|
Placebo Comparator: Placebo
sugar pill
|
Drug: Placebo
Patients will receive a placebo identical in form and dosage to the active drug daily for 16 weeks.
Other Name: sugar pill
|
Detailed Description:
Over the past 20 years, several studies have suggested that sapropterin (tetrahydrobiopterin) might ameliorate core symptoms of autism at least in young (under age 6) subjects. However, those studies had somewhat questionable methodologies, a major one being that the doses of sapropterin used were roughly one tenth that thought to be needed to provide physiologically meaningful increases of sapropterin in the central nervous system (CNS). This study will look at the impact of a sustained exposure to this higher dose in well-diagnosed young children with autism.
Eligibility| Ages Eligible for Study: | 3 Years to 6 Years |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- Parents sign informed consent
- Child meets criteria for autistic disorder (based on score on the Autism Diagnostic Interview—Revised (ADI-R) and the Autism Diagnostic Observation Schedule (ADOS), given by a certified administrator, research reliable)
- Child has a Developmental Quotient (DQ) ≥ 50 (Vineland Adaptive Scales, Interview Edition)
- Parents agree to delay initiation of other treatments during double-blind trial
Exclusion Criteria:
- Child has had seizures in past 6 months or a change in seizure medications in past 4 weeks.
- Child has > 18 points on subscale of (Autism Behavior Checklist) ABC-I
- Child is taking any psychoactive medication other than supplements, anticonvulsants, or soporifics (melatonin, diphenhydramine)
- Child has had any change in standing medications in the past 4 weeks.
- Child has known genetic disorders
- Child has known severe neurological disorders, including cerebral palsy
Contacts and Locations
Please refer to this study by its ClinicalTrials.gov identifier: NCT00850070
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, Ph.D., M.D. | The Children's Health Council |
More Information
No publications provided
| Responsible Party: | Glen R. Elliott, The Children's Health Council |
| ClinicalTrials.gov Identifier: | NCT00850070 History of Changes |
| Other Study ID Numbers: | CHC-0901 |
| Study First Received: | February 20, 2009 |
| Last Updated: | May 11, 2011 |
| Health Authority: | United States: Institutional Review Board |
Keywords provided by The Children's Health Council:
|
autism |
Additional relevant MeSH terms:
|
Autistic Disorder Child Development Disorders, Pervasive Mental Disorders Diagnosed in Childhood Mental Disorders Verapamil Vasodilator Agents Cardiovascular Agents |
Therapeutic Uses Pharmacologic Actions Anti-Arrhythmia Agents Calcium Channel Blockers Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |
ClinicalTrials.gov processed this record on May 19, 2013