Sapropterin as a Treatment for Autistic Disorder

NCT ID: NCT00850070

Last Updated: 2018-05-01

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 46 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2009-03-31
• Study Completion Date: 2011-10-31

Brief Summary

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.

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.

Conditions

Autistic Disorder

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

sapropterin, 100 mg capsules

Sapropterin was supplied as a 100 mg tablet and dosage was based on 20 mg/kg/d, rounding to the nearest 100 mg. Most subjects crushed the tablets and administered it in liquid or a food to mask the taste. Subjects took the same dose daily for 16 weeks.

Group Type: EXPERIMENTAL

sapropterin

Intervention Type: DRUG

Patients will receive sapropterin 20 mg per kilogram per day for 16 weeks

Placebo, matching active drug

The placebo was supplied as a 100 mg tablet, and dosage was based on 20 mg/kg/d, rounding to the nearest 100 mg. Most subjects crushed the tablets and administered it in liquid or a food to mask the taste. Subjects took the same dose daily for 16 weeks.

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

Patients will receive a placebo identical in form and dosage to the active drug daily for 16 weeks.

Interventions

sapropterin

Patients will receive sapropterin 20 mg per kilogram per day for 16 weeks

Intervention Type: DRUG

Placebo

Patients will receive a placebo identical in form and dosage to the active drug daily for 16 weeks.

Intervention Type: DRUG

Other Intervention Names

Kuvan; tetrahydrobiopterin; sugar pill

Eligibility 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

• Minimum Eligible Age: 3 Years
• Maximum Eligible Age: 6 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

BioMarin Pharmaceutical

INDUSTRY

Sponsor Role: collaborator

The Children's Health Council

OTHER

Sponsor Role: lead

Responsible Party

Glen R. Elliott

Chief Psychiatrist and Medical Director

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Glen R Elliott, Ph.D., M.D.

Role: PRINCIPAL_INVESTIGATOR

The Children's Health Council

Locations

The Children's Health Council

Palo Alto, California, United States

Countries

United States

References

Klaiman C, Huffman L, Masaki L, Elliott GR. Tetrahydrobiopterin as a treatment for autism spectrum disorders: a double-blind, placebo-controlled trial. J Child Adolesc Psychopharmacol. 2013 Jun;23(5):320-8. doi: 10.1089/cap.2012.0127.

Reference Type: RESULT

PMID: 23782126 (View on PubMed)

Other Identifiers

CHC-0901

Identifier Type: -

Identifier Source: org_study_id