Study of Acamprosate in Fragile x Syndrome

This study is currently recruiting participants. (see Contacts and Locations)
Verified March 2014 by Children's Hospital Medical Center, Cincinnati
Sponsor:
Collaborator:
Rush University Medical Center
Information provided by (Responsible Party):
Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier:
NCT01911455
First received: July 26, 2013
Last updated: March 5, 2014
Last verified: March 2014
  Purpose

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 Intervention Phase
Fragile X Syndrome
Drug: acamprosate
Drug: Placebo
Phase 2
Phase 3

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Double-Blind, Placebo-Controlled Proof of Concept Study in Youth With Fragile X Syndrome

Resource links provided by NLM:


Further study details as provided by Children's Hospital Medical Center, Cincinnati:

Primary Outcome Measures:
  • Aberrant Behavior Checklist-Social Withdrawal subscale [ Time Frame: Change from baseline to week 10 ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Clinical Global Impressions- Improvement (CGI-I) [ Time Frame: week 10 ] [ Designated as safety issue: No ]
  • Aberrant Behavior Checklist- Hyperactivity (ABC-H) [ Time Frame: Change from baseline to week 10 ] [ Designated as safety issue: No ]
  • Aberrant Behavior Checklist-Social Avoidance (ABC-SA) [ Time Frame: Change from baseline to week 10 ] [ Designated as safety issue: No ]

Estimated Enrollment: 48
Study Start Date: June 2013
Estimated Study Completion Date: September 2016
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
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

  Eligibility

Ages Eligible for Study:   5 Years to 23 Years
Genders Eligible for Study:   Both
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
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01911455

Contacts
Contact: Katie Friedmann, RN 513-636-0523 fragilex@cchmc.org

Locations
United States, Illinois
Rush University Medical Center Recruiting
Chicago, Illinois, United States, 60612
Contact: Jamie Chin    312-942-0025    jamie_a_chin@rush.edu   
Principal Investigator: Elizabeth Berry-Kravis, MD         
United States, Ohio
Cincinnati Children's Hospital Medical Center Recruiting
Cincinnati, Ohio, United States, 45229
Contact: Katie Friedmann, RN    513-636-0523    fragilex@cchmc.org   
Principal Investigator: Craig Erickson, MD         
Sponsors and Collaborators
Children's Hospital Medical Center, Cincinnati
Rush University Medical Center
Investigators
Principal Investigator: Craig Erickson, MD Children's Hospital Medical Center, Cincinnati
Principal Investigator: Elizabeth Berry-Kravis, MD Rush University Medical Center
  More Information

No publications provided

Responsible Party: Children's Hospital Medical Center, Cincinnati
ClinicalTrials.gov Identifier: NCT01911455     History of Changes
Other Study ID Numbers: CIN001- Acamprosate in FX
Study First Received: July 26, 2013
Last Updated: March 5, 2014
Health Authority: United States: Food and Drug Administration
United States: Institutional Review Board

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

Additional relevant MeSH terms:
Fragile X Syndrome
Mental Retardation, X-Linked
Genetic Diseases, X-Linked
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Sex Chromosome Disorders
Chromosome Disorders
Congenital Abnormalities
Genetic Diseases, Inborn
Heredodegenerative Disorders, Nervous System
Acamprosate
Alcohol Deterrents
Central Nervous System Agents
Therapeutic Uses
Pharmacologic Actions

ClinicalTrials.gov processed this record on July 31, 2014