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Study of Acamprosate in Fragile x Syndrome

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ClinicalTrials.gov Identifier: NCT01911455
Recruitment Status : Active, not recruiting
First Posted : July 30, 2013
Last Update Posted : February 11, 2019
Sponsor:
Collaborator:
Rush University Medical Center
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati

Brief Summary:
In this research study we want to understand the effectiveness of a drug treatment, acamprosate, for interfering symptoms (i.e., inattention/hyperactivity, social impairment) associated with Fragile X Syndrome (FXS).

Condition or disease Intervention/treatment Phase
Fragile X Syndrome Drug: acamprosate Drug: Placebo Phase 2 Phase 3

Detailed Description:
Each subject with FXS will receive 10 weeks of blinded treatment with acamprosate or matching placebo. After completion of the double-blind phase, all subjects will have an opportunity to receive acamprosate as part of the study procedures for 16 weeks.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 48 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome
Study Start Date : June 2013
Estimated Primary Completion Date : June 2019
Estimated Study Completion Date : June 2020


Arm Intervention/treatment
Active Comparator: acamprosate
The maximum dose of acamprosate to be used in this study is 666 mg three times daily (total 1998 mg/day) for subjects weight ≥ 50 kg and 1332mg for subjects that weigh < 50 kg.
Drug: acamprosate
Other Name: Campral

Placebo Comparator: Placebo
Placebo will be prescribed with the same frequency and duration as the acamprosate group.
Drug: Placebo



Primary Outcome Measures :
  1. Aberrant Behavior Checklist-Social Withdrawal subscale [ Time Frame: Change from baseline to week 10 ]
    The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.


Secondary Outcome Measures :
  1. Clinical Global Impressions- Improvement (CGI-I) [ Time Frame: week 10 ]
    The CGI-I global improvement item is a 7-point Likert scale designed to measure symptomatic change at a specific time as compared to baseline. CGI-I is a gold standard global measure of potential change with treatment in placebo-controlled pharmacotherapy trials in developmental disabilities.

  2. Aberrant Behavior Checklist- Hyperactivity (ABC-H) [ Time Frame: Change from baseline to week 10 ]
    The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.

  3. Aberrant Behavior Checklist-Social Avoidance (ABC-SA) [ Time Frame: Change from baseline to week 10 ]
    The ABC is the gold standard parent/caregiver reported behavioral outcome measure for use in developmental disability clinical trials.



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Ages Eligible for Study:   5 Years to 23 Years   (Child, Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Diagnostic confirmation of full mutation FXS
  • Age ≥5 years and <23 years
  • General good health as determined by physical exam, medical history and laboratory work up.

Exclusion Criteria:

  • Use of more than two psychotropic medications (medications affecting behavior).
  • Unstable dosing of any psychotropic medication (medication affecting behavior)
  • Problems with kidney functioning
  • Unstable seizure disorder
  • Change in any anti-convulsant drug dosing in the 60 days prior to study entry
  • Prior adequate treatment trial with acamprosate as determined by the study doctor
  • Pregnant or lactating females

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT01911455


Locations
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United States, Illinois
Rush University Medical Center
Chicago, Illinois, United States, 60612
United States, Ohio
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Rush University Medical Center
Investigators
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Principal Investigator: Craig Erickson, MD Children's Hospital Medical Center, Cincinnati
Principal Investigator: Elizabeth Berry-Kravis, MD Rush University Medical Center

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Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01911455     History of Changes
Other Study ID Numbers: CIN001- Acamprosate in FX
First Posted: July 30, 2013    Key Record Dates
Last Update Posted: February 11, 2019
Last Verified: February 2019

Keywords provided by Children's Hospital Medical Center, Cincinnati:
Fragile X Syndrome

Additional relevant MeSH terms:
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Acamprosate
Syndrome
Fragile X Syndrome
Disease
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
Alcohol Deterrents