A Prospective Open-label Study of Aripiprazole in Fragile X Syndrome

NCT00420459 | Completed Phase 2 Results DMC

• 1 other identifier: 0609-22
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine the effectiveness and tolerability of aripiprazole in the treatment of children and adolescents with Fragile X Syndrome.

Conditions

Fragile X Syndrome

Outcome Measures

Primary Outcomes (2)

• Aberrant Behavior Checklist

  The Aberrant Behavior Checklist (ABC) is a 58-item measure of maladaptive behaviors and is used as a measure of drug effects. The ABC has 5 subscales: Social Withdrawal (16 items) ranging from 0 (not at all) to 48 (severe), Irritability (15 items) ranging from 0 (not at all) to 45 (severe), Inappropriate Speech (4 items) ranging from 0 (not at all) to 12 (severe), Hyperactivity (16 items) ranging from 0 (not at all) to 48 (severe), and Stereotypy (7 items) ranging from 0 (not at all) to 21 (severe). Items are rated from 0 (not at all) to 3 (severe).

  Obtained at Baseline and Week 12

• Clinical Global Impressions- Severity

  The Clinical Global Impression - Severity scale (CGI-S) is a 7-point scale that requires the clinician to rate the severity of the patient's illness at the time of assessment, relative to the clinician's past experience with patients who have the same diagnosis. Considering total clinical experience, a patient is assessed on severity of mental illness at the time of rating 1, normal, not at all ill; 2, borderline mentally ill; 3, mildly ill; 4, moderately ill; 5, markedly ill; 6, severely ill; or 7, extremely ill.

  Obtained at Baseline and Week 12

Secondary Outcomes (4)

• The Children's Yale-Brown Obsessive Compulsive Scale

  Obtained at Baseline and Week 12

• Social Responsiveness Scale

  Obtained at Baseline and Week 12

• The Vineland Adaptive Behavior Scales

  Screen Visit

• The Vineland Maladaptive Behavior Subscales

  Week 12

Study Arms (1)

Aripiprazole

EXPERIMENTAL

Drug: Aripiprazole

Interventions

Aripiprazole DRUG

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.

Also known as: Abilify

Aripiprazole

Eligibility Criteria

• Age: 5 Years - 35 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64)

You may qualify if:

• Males and females between the ages of 5 and 35 years and
• Body weight greater than or equal to 15 kg
• Confirmed diagnosis of Fragile X Syndrome based upon genetic testing results.
• Outpatients.
• 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.
• Clinical Global Impression Scale Severity score (CGI-S) of at greater than or equal to 4 (Moderately Ill)
• A score of at greater than or equal to 18 on the Irritability subscale of the Aberrant Behavior Checklist (ABC) at screen and baseline.
• Mental age of greater than or equal to 18 months as measured by the Wechsler, revised Leiter, or Mullen tests
• 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.

You may not qualify if:

• DSM-IV diagnosis of schizophrenia, another psychotic disorder, bipolar disorder or alcohol or other substance abuse within the last 6 months.
• 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.
• Subjects with an unstable seizure disorder will be excluded.
• Females with a positive urine pregnancy test.
• 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.
• Evidence of hypersensitivity to aripiprazole (defined as an allergic response \[e.g., skin rash\] or potentially serious adverse effect \[e.g., significant tachycardia\]).
• History of neuroleptic malignant syndrome.
• 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.

Sponsors & Collaborators

• Indiana University School of Medicine: lead

Study Sites (1)

Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children

Indianapolis, Indiana, 46202, United States

Location

Related Publications (1)

• Erickson CA, Stigler KA, Wink LK, Mullett JE, Kohn A, Posey DJ, McDougle CJ. A prospective open-label study of aripiprazole in fragile X syndrome. Psychopharmacology (Berl). 2011 Jul;216(1):85-90. doi: 10.1007/s00213-011-2194-7. Epub 2011 Feb 12.

  PMID: 21318565 BACKGROUND

MeSH Terms

Conditions

Fragile X Syndrome

Interventions

Aripiprazole

Condition Hierarchy (Ancestors)

X-Linked Intellectual Disability; Intellectual Disability; Neurobehavioral Manifestations; Neurologic Manifestations; Nervous System Diseases; Sex Chromosome Disorders; Chromosome Disorders; Congenital Abnormalities; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, Inborn; Genetic Diseases, X-Linked; Heredodegenerative Disorders, Nervous System

Intervention Hierarchy (Ancestors)

Piperazines; Heterocyclic Compounds, 1-Ring; Heterocyclic Compounds; Quinolones; Quinolines; Heterocyclic Compounds, 2-Ring; Heterocyclic Compounds, Fused-Ring

Results Point of Contact

• Title: Craig Erickson
• Organization: Cincinnati Childrens Hospital

craig.erickson@cchmc.org

5136366265

Study Officials

• Craig A. Erickson, MD

  Indiana University

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 9, 2007
• First Posted: January 11, 2007
• Study Start: April 1, 2007
• Primary Completion: March 1, 2010
• Study Completion: March 1, 2010
• Last Updated: April 18, 2017
• Results First Posted: April 18, 2017

Record last verified: 2017-03

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)