Open-Label Extension Study of Kuvan for Autism

The Children's Health Council

Status and phase: Completed; Phase 3; Phase 2

Conditions: Autistic Disorder

Treatments: Drug: Kuvan®

Study type: Interventional

Funder types: Other; Industry

Identifiers

NCT00943579

CHC-0902

Details and patient eligibility

About

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.

Full description

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.

Enrollment: 41 patients

Sex: All

Ages: 3 to 6 years old

Volunteers: No Healthy Volunteers

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)

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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41 participants in 1 patient group

Kuvan®

Experimental group

Description:

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

Treatment:

Drug: Kuvan®