Phase 2
N=46
Sapropterin as a Treatment for Autistic Disorder
Autistic Disorder
Bottom Line
View on ClinicalTrials.gov: NCT00850070 ↗Enrolled (actual)
46
Serious AEs
6.5%
Results posted
Feb 2014
Primary outcome: Primary: Clinical Global Impression -- Improvement (CGI-I) Scale — 5; 3 participants — p=<.35
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- sapropterin (Drug); Placebo (Drug)
- Age
- Pediatric · 3+ yrs
- Sex
- All
- Sponsor
- The Children's Health Council
- Primary completion
- Aug 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Clinical Global Impression -- Improvement (CGI-I) Scale |
5; 3 | <.35 |
| PRIMARY Clinical Global Impression -- Severity (CGI-S) Scale |
3; 1 | <0.06 |
| SECONDARY Preschool Language Scale-Fourth Edition (PLS-4). Assesses Expressive and Receptive Language Skills in Ages Birth Through 6 Years, 11 Months. |
84; 60 | — |
| SECONDARY Vineland Adaptive Behavior Scale-II. |
344.76; 294.9 | — |
| SECONDARY Children's Yale Brown Obsessive Compulsive Scale (C-YBOCS) |
— | — |
| SECONDARY Connor's Preschool ADHD Questionnaire |
— | — |
| SECONDARY Adverse Events Scale |
— | — |
| SECONDARY Aberrant Behavior Checklist (ABC) - Inappropriate Speech |
2.6; 3.9 | 0.05 |
| SECONDARY Social Responsiveness Scale (SRS) |
76.7; 83.2 | — |
| SECONDARY Parent Global Assessment (PGA) Scale |
— | — |
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.
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
Data sourced from ClinicalTrials.gov (NCT00850070). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.