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Aripiprazole in Fragile X Syndrome

This study has been completed.
Information provided by (Responsible Party):
Indiana University ( Indiana University School of Medicine ) Identifier:
First received: January 9, 2007
Last updated: April 25, 2012
Last verified: April 2012
The purpose of this study is to determine the effectiveness and tolerability of aripiprazole in the treatment of children and adolescents with FXS. We hypothesize that aripiprazole will be effective in decreasing aggression, SIB, agitation, and interfering repetitive behavior commonly observed in individuals with FXS. We also hypothesize that aripiprazole will be well tolerated.

Condition Intervention Phase
Fragile X Syndrome
Behavioral Problems Associated With Fragile X Syndrome
Drug: Aripiprazole
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: Aripiprazole in Fragile X Syndrome

Resource links provided by NLM:

Further study details as provided by Indiana University:

Primary Outcome Measures:
  • Parent-rated irritability subscale of Aberrant Behavior Checklist (ABC) [ Time Frame: Completed at every visit ]
  • Clinician-rated Clinical Global Impression (CGI)- global improvement item (CGI-I) [ Time Frame: Completed at every visit ]

Secondary Outcome Measures:
  • Aberrant Behavior Checklist sub-scales II, III, IV &V [ Time Frame: Completed at every visit ]
  • The Vineland Adaptive Behavior Scales [ Time Frame: Screen Visit ]
  • The Vineland Maladaptive Behavior Subscales [ Time Frame: Every 4 weeks ]
  • The Children's Yale-Brown Obsessive Compulsive Scale [ Time Frame: At baseline and every 4 weeks. ]

Enrollment: 12
Study Start Date: April 2007
Study Completion Date: March 2010
Primary Completion Date: March 2010 (Final data collection date for primary outcome measure)
Intervention Details:
    Drug: Aripiprazole
    All subjects will initially receive 2.5 mg/day of aripiprazole during the first week. The dosage may be increased to a maximum of 20 mg/day over 8 weeks.
    Other Name: Abilify

Ages Eligible for Study:   5 Years to 35 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Males and females between the ages of 5 and 35 years and
  2. Body weight greater than or equal to 15 kg
  3. Confirmed diagnosis of Fragile X Syndrome based upon genetic testing results.
  4. Outpatients.
  5. Psychotropic medication-free for at least 2 weeks prior to screening laboratory tests and electrocardiogram. (Except 5 weeks for fluoxetine and 4 weeks for all typical and atypical antipsychotics that have been administered for at least a 4 week period.) Exceptions to medication-free status will include drugs given at bedtime targeting insomnia. Such drugs may include melatonin, clonidine, chloral hydrate, diphenhydramine, ramelteon, benzodiazepines, or other sedative-hypnotics.
  6. Clinical Global Impression Scale Severity score (CGI-S) of at greater than or equal to 4 (Moderately Ill)
  7. A score of at greater than or equal to 18 on the Irritability subscale of the Aberrant Behavior Checklist (ABC) at screen and baseline.
  8. Mental age of greater than or equal to 18 months as measured by the Wechsler, revised Leiter, or Mullen tests
  9. Each subject must be in good physical health as determined by screening procedures which will include a detailed medical history, complete physical and neurological examination.

Exclusion Criteria:

  1. DSM-IV diagnosis of schizophrenia, another psychotic disorder, bipolar disorder or alcohol or other substance abuse within the last 6 months.
  2. A significant medical condition such as heart, liver, renal or pulmonary disease, or an actively treated seizure disorder, as determined by history, physical examination or laboratory testing.
  3. Subjects with an unstable seizure disorder will be excluded.
  4. Females with a positive urine pregnancy test.
  5. Evidence of a prior adequate trial of aripiprazole (defined as a duration of greater than or equal to 2 weeks at a dose of at least 5 mg per day). When there is not evidence of a prior adequate trial of aripiprazole, subjects must be medication-free for at least 2 weeks prior to baseline.
  6. Evidence of hypersensitivity to aripiprazole (defined as an allergic response [e.g., skin rash] or potentially serious adverse effect [e.g., significant tachycardia]).
  7. History of neuroleptic malignant syndrome.
  8. Subjects who, in the opinion of the investigator, are unsuitable in any other way to participate in this study including being unable to comply with the requirements of the study for any reason.
  Contacts and Locations
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Please refer to this study by its identifier: NCT00420459

Sponsors and Collaborators
Indiana University School of Medicine
Principal Investigator: Christopher J McDougle, MD Indiana University
  More Information

Responsible Party: Indiana University School of Medicine Identifier: NCT00420459     History of Changes
Other Study ID Numbers: 0609-22
Study First Received: January 9, 2007
Last Updated: April 25, 2012

Additional relevant MeSH terms:
Fragile X Syndrome
Problem Behavior
Pathologic Processes
Mental Retardation, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Genetic Diseases, X-Linked
Heredodegenerative Disorders, Nervous System
Behavioral Symptoms
Antipsychotic Agents
Tranquilizing Agents
Central Nervous System Depressants
Physiological Effects of Drugs
Psychotropic Drugs processed this record on March 22, 2017