NCT00943579

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.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
41

participants targeted

Target at P25-P50 for phase_2

Timeline
Completed

Started Aug 2009

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

July 20, 2009

Completed
2 days until next milestone

First Posted

Study publicly available on registry

July 22, 2009

Completed
10 days until next milestone

Study Start

First participant enrolled

August 1, 2009

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 1, 2011

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 1, 2012

Completed
1.3 years until next milestone

Results Posted

Study results publicly available

July 4, 2013

Completed
Last Updated

May 2, 2018

Status Verified

April 1, 2018

Enrollment Period

2.3 years

First QC Date

July 20, 2009

Results QC Date

January 16, 2013

Last Update Submit

April 30, 2018

Conditions

Autistic Disorder

Keywords

autismautistic disordertetrahydrobiopterinsapropterintreatment

Outcome Measures

Primary Outcomes (1)

  • Clinical Global Impressions Scale

    This is a summary judgment made by a trained clinician based on observed and reported behaviors of the child compared to baseline. It is a 7-point scale (1) very much improved, (2) much improved, (3) minimally improved (4) no change, (5) minimally worse, (6) much worse and (7) very much worse. Chi-square analyses were used to assess change in CHI-I scores (by group, post-test)Mixed-effects regression models determined the main effects attributed to differences by group (BH4 and placebo), time (treated as categorical at levels baseline, 8 weeks, and 16 weeks) and the group-by-time interaction. The mixed-effects models accounted for each participant's outcome data at each time point. The mixed-effects regression model is robust to data dependency that occurs with the repeated assessments of individuals over time \& can handle missing data. We used random intercept and trend modeling that accounts for each individual's initial level of symptom severity/functioning and rate of change/time

    16 weeks

Secondary Outcomes (7)

  • Vineland Adaptive Behavior Scale, 2nd Edition

    Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902).

  • Children's Yale Brown Obsessive Compulsive Scale

    Weeks 8 & 16

  • Parental Global Assessment

    Weeks 8 & 16

  • Preschool Language Scale, 4th Edition (PLS-4)

    Weeks baseline (week 16 from CHC-0901), 8 and 16. Primary outcome assessment looked at change between baseline (week 16 from CHC-0901 and week 16 of CHC-0902).

  • Connor's Preschool ADHD Questionnaire

    Weeks 8 & 16

  • +2 more secondary outcomes

Study Arms (1)

Kuvan®

EXPERIMENTAL

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

Drug: Kuvan®

Interventions

Kuvan®DRUG

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

Also known as: sapropterin, tetrahydrobiopterin
Kuvan®

Eligibility Criteria

Age3 Years - 6 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

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

You may not qualify if:

  • Child failed to complete CHC 0901 (NCT00850070)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

The Children's Health Council

Palo Alto, California, 94304, United States

Location

MeSH Terms

Conditions

Autistic Disorder

Interventions

sapropterin

Condition Hierarchy (Ancestors)

Autism Spectrum DisorderChild Development Disorders, PervasiveNeurodevelopmental DisordersMental Disorders

Results Point of Contact

Title
Glen R. Elliott, Ph.D., MD
Organization
Children's Health Council
gelliott@chconline.org
650.688.3649

Study Officials

  • Glen R Elliott, PhD, MD

    The Children's Health Council

    PRINCIPAL INVESTIGATOR

Publication Agreements

PI is Sponsor Employee
Yes
Restrictive Agreement
No

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Chief Psychiatrist and Medical Director

Study Record Dates

First Submitted

July 20, 2009

First Posted

July 22, 2009

Study Start

August 1, 2009

Primary Completion

December 1, 2011

Study Completion

March 1, 2012

Last Updated

May 2, 2018

Results First Posted

July 4, 2013

Record last verified: 2018-04

Locations

US(1)