Clinical Trial of Aripiprazol in Fragile X Syndrome

With a FRAXA Research Foundation grant in 2006, Dr. Erickson conducted a pilot clinical trial of an available medicine, aripiprazole (brand-name Abilify). This was an open-label 12-week trial in 12 people aged 6–25 years with fragile X. Results were promising and published: 10 of the 12 participants showed behavioral improvements.

Craig Erickson, MD, Cincinnati Children's Hospital
$30,000 Grant
Craig Erickson, PMD
Principal Investigator
Indiana University School of Medicine
2006 FRAXA Research Grant
$30,000

People with fragile X syndrome sometimes exhibit irritable behavior marked by aggression, self-injury, and severe tantrums. Despite frequent clinical use of atypical antipsychotic drugs to target this behavioral cluster, no systematic trials to date have assessed the efficacy and safety of these drugs in persons with fragile X.

We conducted a prospective open-label 12-week trial of aripiprazole in 12 persons aged 6-25 years (mean age, 14.3 years) with fragile X syndrome who were free of other psychoactive drugs.

Aripiprazole use (mean dose, 9.8 mg/day) was associated with treatment response (defined by a Clinical Global Impressions-Improvement scale score of much improved or very much improved and a = 25% improvement on the Aberrant Behavior Checklist-Irritability subscale) in 10 of 12 (87%) people. Two individuals (13%) discontinued aripiprazole prior to study completion due to adverse events. One discontinuation was due to akathisia, mild drooling, and mild tiredness, and the other due to moderate tiredness and moderate drooling. No significant changes in vital signs including weight or laboratory measures occurred during treatment with aripiprazole.

Aripiprazole was generally safe and well tolerated and was associated with significant improvement in irritable behavior. Given these findings, a double-blind, placebo-controlled study of aripiprazole in fragile X syndrome is warranted.

Results Published in 2011: A prospective open-label study of aripiprazole in fragile X syndrome.

Christopher McDougle, MD
Co-Principal Investigator

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