Trial Outcomes & Findings for Pharmacogenomics in Autism Treatment (NCT NCT00584701)

Results Overview

Aberrant Behavior Checklist-Irritability (ABC-I)subscale: measure of assessing changes in symptoms of irritability in children with autism (survey that was normed on a developmentally delayed population of children and adults and is usually completed by a parent or caregiver. There are 45 items that are rated on a 4-point scale from "no problem" to "major problem." ABC-I scores ranges from 0 (best) to 45 (worst). A negative change signifies improvement. We measured percent change of ABC-I scores from 8 weeks after risperidone treatment compared to baseline.

Recruitment status

COMPLETED

Study phase

PHASE2/PHASE3

Target enrollment

49 participants

Primary outcome timeframe

Baseline, 8 weeks

Results posted on

2012-07-24

Participant Flow

Participants recruitment at the UCDavis MIND Institute from the local community.

Participant milestones

Participant milestones
Measure	Active Risperidone Risperidone was started at 0.5mg at bedtime for 4 days. If that dosage was tolerated and there were continued behavioral symptoms, the dose was increased to 1mg at bedtime for an additional 4 days. If tolerated and indicated, 0.5mg was added in the morning for a daily total of 1.5 mg.
Active Risperidone STARTED	49
Active Risperidone COMPLETED	46
Active Risperidone NOT COMPLETED	3
Follow-up STARTED	46
Follow-up COMPLETED	34
Follow-up NOT COMPLETED	12

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Pharmacogenomics in Autism Treatment

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	Active Risperidone n=49 Participants Risperidone was started at 0.5mg at bedtime for 4 days. If that dosage was tolerated and there were continued behavioral symptoms, the dose was increased to 1mg at bedtime for an additional 4 days. If tolerated and indicated, 0.5mg was added in the morning for a daily total of 1.5 mg.
Age, Categorical <=18 years	49 Participants n=5 Participants
Age, Categorical Between 18 and 65 years	0 Participants n=5 Participants
Age, Categorical >=65 years	0 Participants n=5 Participants
Age Continuous	9.4 years STANDARD_DEVIATION 4.3 • n=5 Participants
Sex: Female, Male Female	9 Participants n=5 Participants
Sex: Female, Male Male	40 Participants n=5 Participants
Region of Enrollment United States	49 participants n=5 Participants

PRIMARY outcome

Timeframe: Baseline, 8 weeks

Aberrant Behavior Checklist-Irritability (ABC-I)subscale: measure of assessing changes in symptoms of irritability in children with autism (survey that was normed on a developmentally delayed population of children and adults and is usually completed by a parent or caregiver. There are 45 items that are rated on a 4-point scale from "no problem" to "major problem." ABC-I scores ranges from 0 (best) to 45 (worst). A negative change signifies improvement. We measured percent change of ABC-I scores from 8 weeks after risperidone treatment compared to baseline.

Outcome measures

Outcome measures
Measure	Active Risperidone n=49 Participants Risperidone was started at 0.5mg at bedtime for 4 days. If that dosage was tolerated and there were continued behavioral symptoms, the dose was increased to 1mg at bedtime for an additional 4 days. If tolerated and indicated, 0.5mg was added in the morning for a daily total of 1.5 mg.
Percent Change of ABC - Irritability Subscale Score	-44.5 percent change in scores Interval -95.0 to 6.0

SECONDARY outcome

Timeframe: Baseline, 8 Weeks

Affymetrix GeneChip Human Exon 1.0 ST Arrays (Affymetrix, Santa Clara, CA, USA) were used to obtain gene expression values. Raw data (Affymetrix.CEL files) was imported into Partek Genomics Suite 6.4 (Partek, St Louis, MO, USA). Probe summarization and probe set normalization were performed using robust multichip average, which included background correction, quantile normalization, log2 transformation and median polish probe set summarization. Exons in genes correlated with percentage improvement on the Aberrant Behavior Checklist Irritability subscale were identified.

Outcome measures

Outcome measures
Measure	Active Risperidone n=49 Participants Risperidone was started at 0.5mg at bedtime for 4 days. If that dosage was tolerated and there were continued behavioral symptoms, the dose was increased to 1mg at bedtime for an additional 4 days. If tolerated and indicated, 0.5mg was added in the morning for a daily total of 1.5 mg.
Exon Expression Positively or Negatively Correlated With Percentage Improvement in ABC-I	5 Number of Correlated Genes

Adverse Events

Active Risperidone

Serious events: 0 serious events

Other events: 0 other events

Deaths: 0 deaths

Serious adverse events

Adverse event data not reported

Other adverse events

Adverse event data not reported

Additional Information

Robert Hendren, DO

UCSF

Phone: 415-476-7198

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place