Biomarkers in Autism of Aripiprazole and Risperidone Treatment (BAART)

Medical University of South Carolina (MUSC)

Status and phase

Completed

Phase 4

Conditions

Autistic Disorder

Treatments

Drug: Risperidone

Drug: Aripiprazole

Study type

Interventional

Funder types

Other

Identifiers

NCT01333072

R01HD062550-01A1

Details and patient eligibility

About

Full description

The Biomarkers in autism of aripiprazole and risperidone treatment (BAART) project will provide evidence-based guidance in the selection and monitoring of drug treatment of autism. BAART involves 3 academic centers across South Carolina with expertise in phenotyping patients with autistic spectrum disorders, assessing patient response in clinical trials, and expertise in pharmacogenomic research. Although the FDA has approved use of the antipsychotic drugs risperidone and aripiprazole for irritability associated with autistic disorder, a moderate response rate in pivotal clinical trials and concerns over tolerability and weight gain can force clinicians to select alternative drug treatments for which evidence-based support is sparse. BAART will assess predictors of efficacy, tolerability, and safety in 200 children 6-17 years old with autistic disorder (AD) during a double-blind, randomized 10 week treatment period with either risperidone or aripiprazole. Responders who complete the study may continue with medication treatment for three months. Factors considered will include 1) psychiatric history; 2) symptom response; 3) psychosocial support; 4) measures of tolerability; 5) serum prolactin and brain-derived neurotrophic factor concentration; and 5) a variety of single nucleotide polymorphisms related to target genes for drug disposition and transport, response, and tolerability. The BAART project will result in evidence-based guidelines for selection and monitoring of drug treatment of children and adolescents with AD.

Enrollment

80 patients

Sex

All

Ages

6 to 17 years old

Volunteers

No Healthy Volunteers

Inclusion and exclusion criteria

Inclusion Criteria

Aged 6 to 17 years and weight of at least 15 kg
Meet DSM-IV criteria for of AD, established by chart review, clinical judgment and the Autism Diagnostic Interview- Revised (ADI-R) criteria
Clinical Global Impressions Severity (CGI-S) score of >4 (moderately ill)
ABC Irritability subscale score of >18
Mental age of at least 18 months
If female and sexually active, must agree to an acceptable method of birth control during the trial
Medication free or adequate washout period (2-4 weeks prior to enrollment) of psychoactive drugs (anticonvulsants permitted for seizure management if dosage is stable for 4 weeks)
Parent/guardian able to read and provide informed consent.

Exclusion Criteria

Psychiatric disorder that is effectively managed by psychoactive medication (e.g. ADHD, MDD)
Prior diagnosis or evidence of genetic or other disorder that may interfere with assessments (e.g. Fragile X syndrome, Fetal alcohol syndrome, history of very low birth weight) assessed by personal and family history, dysmorphology, and clinical judgment.
Prior use of risperidone or aripiprazole for more than 2 weeks
Seizure during the past 6 months
History or evidence of a medical condition that would expose them to an undue risk of a significant adverse event or interfere with assessments during the trial including but not limited to hepatic, renal, respiratory, cardiovascular, endocrine, hematologic or immunologic disease as determined by the clinical judgment of the investigator
Current suicidal or homicidal risk
Positive urine pregnancy test at baseline
Dependent on other substances, with the exception of nicotine or caffeine