Open-Label Extension Study of Kuvan for Autism

NCT ID: NCT00943579

Last Updated: 2018-05-02

Study Results

Results available

Outcome measurements, participant flow, baseline characteristics, and adverse events have been published for this study.

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Basic Information

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Recruitment Status

COMPLETED

Clinical Phase

PHASE2/PHASE3

Total Enrollment

41 participants

Study Classification

INTERVENTIONAL

Study Start Date

2009-08-31

Study Completion Date

2012-03-31

Brief Summary

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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.

Detailed Description

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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.

Conditions

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Autistic Disorder

Study Design

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Allocation Method

NA

Intervention Model

SINGLE_GROUP

Primary Study Purpose

TREATMENT

Blinding Strategy

NONE

Study Groups

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Kuvan®

Kuvan® (sapropterin) will be administered to all subjects at 20 mg/kg/day for 16 weeks.

Group Type EXPERIMENTAL

Kuvan®

Intervention Type DRUG

Brand-name Kuvan® (sapropterin) will be administered to all subjects at a dose of 20 mg/kg/day for 16 weeks.

Interventions

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Kuvan®

Brand-name Kuvan® (sapropterin) will be administered to all subjects at a dose of 20 mg/kg/day for 16 weeks.

Intervention Type DRUG

Other Intervention Names

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sapropterin tetrahydrobiopterin

Eligibility Criteria

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Inclusion Criteria

* All subjects must have completed earlier trial, CHC 0901 (NCT00850070)
* Parents must be willing and able to sign informed consent

Exclusion Criteria

* Child failed to complete CHC 0901 (NCT00850070)
Minimum Eligible Age

3 Years

Maximum Eligible Age

6 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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BioMarin Pharmaceutical

INDUSTRY

Sponsor Role collaborator

The Children's Health Council

OTHER

Sponsor Role lead

Responsible Party

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Glen R. Elliott

Chief Psychiatrist and Medical Director

Responsibility Role PRINCIPAL_INVESTIGATOR

Principal Investigators

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Glen R Elliott, PhD, MD

Role: PRINCIPAL_INVESTIGATOR

The Children's Health Council

Locations

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The Children's Health Council

Palo Alto, California, United States

Site Status

Countries

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United States

Other Identifiers

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CHC-0902

Identifier Type: -

Identifier Source: org_study_id

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