Phase 3
N=49
Pharmacogenomics in Autism Treatment
Autism
Bottom Line
View on ClinicalTrials.gov: NCT00584701 ↗Enrolled (actual)
49
Serious AEs
0.0%
Results posted
Jul 2012
Primary outcome: Primary: Percent Change of ABC - Irritability Subscale Score — -44.5 percent change in scores
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Risperidone (Drug)
- Age
- Pediatric, Adult · 4+ yrs
- Sex
- All
- Sponsor
- University of California, San Francisco
- Primary completion
- May 2009
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change of ABC - Irritability Subscale Score |
-44.5 | — |
| SECONDARY Exon Expression Positively or Negatively Correlated With Percentage Improvement in ABC-I |
5 | <.001 sig |
Summary
Autism is a complex neurodevelopmental disorder that is thought to involve an interaction between multiple and variable susceptibility genes, environmental factors, and epigenetic effects. Great concern has been raised about the marked increase in the prevalence of autism spectrum disorders in the last decade. Risperidone, the most studied atypical antipsychotic used in children, has been shown to improve severe behavioral difficulties in over half of children with autism who have these difficulties. However, not all children with autism and severe behavioral problems respond to risperidone, and for a few, it has significant side effects.
Two controlled studies and numerous open-label and long term studies in children with autism spectrum disorders using the atypical antipsychotic risperidone show a significant decrease of associated serious behavioral problems. The use of atypical antipsychotics is of great concern, however, because of their significant side effects and the fact that only two-thirds of children positively respond. Ways to predict response, appropriate dosage and serious side effects are needed.
Eligibility Criteria
Inclusion Criteria
- Willingness to participate and written informed consent obtained from parent and when indicated by subject
- Confirmed DSM-IV-TR diagnosis of Autistic Disorder or Asperger's Disorder using the ADOS and the ADI-R, the current gold standards for diagnosing autism spectrum disorders, subject history, and clinical consensus with PI. The ADI-R and ADOS will be administered by research-reliable clinicians, including a clinical psychologist, a certified trainer on the ADOS and the ADI-R
- rated by study clinician as at least "moderate" on the CGI-Severity scale (a rating of ≥ 4) and greater than 18 on the ABC Irritability subscale
Males or females of any race between 4 and 18 years of age (5) A nonverbal IQ greater than or equal to 55 on the Stanford-Binet:V (6) Women of childbearing potential must use an adequate method of contraception throughout the study.
Exclusion Criteria
- Primary diagnosis of bipolar disorder, schizophrenia, or autism spectrum disorder other than Autistic and Asperger's Disorders
- Nonverbal IQ lower than 55 (Stanford-Binet:V)
- History of seizure activity in the past year (active seizures might confuse efficacy ratings)
- fever, infection, metabolic disturbance or any severe medical illness in the past year
- typical or atypical antipsychotic use within 8 weeks of study entry
- Inability of parents or care takers to give informed consent, travel to the visits, administer medication, or arrange for completion of rating scales. Other non-antipsychotic medications and non-pharmacological treatments will be allowed if started at least 2 months prior to the initial screening and must remain constant for the 8 weeks of this study. These treatments will be recorded as will a history of past trials of medications. Prohibiting ongoing treatment would be difficult to justify to parents and to the IRB and would make adequate recruitment for this pilot study difficult.
Data sourced from ClinicalTrials.gov (NCT00584701). 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.