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Acamprosate in Youth With Fragile X Syndrome

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
Indiana University
ClinicalTrials.gov Identifier:
NCT01300923
First received: August 25, 2010
Last updated: April 27, 2017
Last verified: April 2017
  Purpose
Fragile X syndrome (FXS) is the most common inherited form of developmental disability. FXS is inherited from the carrier parent, most often the mothers. FXS is associated with severe interfering behavioral symptoms which include anxiety related symptoms, attention deficit hyperactivity, and aggressive behaviors. Approximately 25-33% of individuals with FXS also meet criteria for autistic disorder. The hypothesis of this study is that treatment with acamprosate will reduce inattention/hyperactivity, language impairment, irritability, social deficits, and cognitive delay in youth with FXS. The purpose of this study is to investigate the effectiveness and tolerability of acamprosate in youth with Fragile X Syndrome and to assess the potential psychophysiological differences between FXS and autism spectrum disorders.

Condition Intervention Phase
Fragile X Syndrome
Autism Spectrum Disorders
Drug: Acamprosate
Phase 3

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: No masking
Primary Purpose: Treatment
Official Title: Pilot Study of Acamprosate in Youth With Fragile X Syndrome

Resource links provided by NLM:


Further study details as provided by Indiana University:

Primary Outcome Measures:
  • Clinical Global Impression- Severity Scale (CGI-S) [ Time Frame: Week 10 ]
    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.


Secondary Outcome Measures:
  • The Aberrant Behavior Checklist (ABC) [ Time Frame: Week 10 ]
    The Aberrant Behavior Checklist (ABC) is a 58-item rating scale used to assess maladaptive behaviors across five original subscales: Irritability (15 items from 0-45), Social Withdrawal (16 items from 0-48), Stereotypy (7 items from 0-21), Hyperactivity (16 items from 0-48), Inappropriate Speech (4 items from 0-12). Additionally, Social Avoidance, a newly developed four-item subscale (from 0-12) of the ABC that captures core social avoidance aspects of Fragile X Syndrome is reported. All items on the ABC are rated from 0 (not at all a problem) to 3 (the problem is severe in degree). Higher scores indicate greater maladaptive behaviors. Differences between Baseline and Week 10 are used as an indicator of change.

  • Social Responsiveness Scale [ Time Frame: Week 10 ]
    The 65-item SRS is a standardized measure of the core symptoms of autism. Each item is scored on a 4-point Likert scale. The score of each individual item is summed to create a total raw score. A total scores results are as follows: 0-62: Within normal limits 63-79: Mild range of impairment 80-108: Moderate range of impairment 109-149: Severe range of impairment

  • Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD [ Time Frame: Week 10 ]
    The Children's Yale-Brown Obsessive Compulsive Scales-Modified (CY-BOCS) is a 5-item, semi-structured clinician rating scale modified designed to rate the current severity of repetitive behavior in children and adolescents with PDD. Once the current repetitive behaviors are identified, they are separately rated on 5 items: Time Spent, Interference, Distress, Resistance, and Control. Each of these items is scored on a 5-point scale form 0 (least symptomatic) to 4 (most symptomatic). The CY-BOCS yields a Total Score from 0 to 20 and is sensitive to change.

  • ADHD Rating Scale 4th Edition [ Time Frame: Week 10 ]
    The ADHD Rating Scale is an 18-item scale directly derived from DSM-IV criteria for Attention Deficit Hyperactivity Disorder. The ADHD Rating Scale-IV is completed by the parent and scored by a clinician. The scale consists of 2 subscales: inattention (9 items) and hyperactivity-impulsivity (9 items). If 3 or more items are skipped, the clinician should use extreme caution in interpreting the scale. The total score can range from 0 to 54, with a higher score indicating greater severity.

  • Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain [ Time Frame: Week 10 ]
    The VABS-II is a semi-structured interview designed to assess adaptive functioning in communication, daily living, socialization and motor skills. Recognizing that language is a major area of impairment in the study population, the Communication Domain (99 Items from 0-198), in particular the Expressive Subdomain (54 Items from 0-108) are of interest in this study. Items arranged in a developmental sequence are rated on a 3-point scale. Each item is scored from 0 (never performs the behavior) to 3 (usually performs the behavior independently). Higher scores indicate higher adaptive functioning. Differences between Baseline and Week 10 are used as an indicator of change.

  • Peabody Picture Vocabulary [ Time Frame: Week 10 ]
    The Peabody Picture Vocabulary Test is one of the most commonly used assessment tests that measure verbal ability in standard American English vocabulary. This test has been nationally standardized using examinees from various age groups, from children to adults. Thus, the raw scores are equated to mental age, using the norms obtained from standardization. The total standard scores range from 40 (worse receptive vocabulary) to 160 (better receptive vocabulary). The scores can also be converted to percentile rank.

  • Brain-derived Neurotrophic Factor (BDNF) [ Time Frame: Screen and Week 10 ]
    BDNF is a protein that supports the survival of existing neurons and growth and differentiation of new neurons and synapses.


Enrollment: 14
Study Start Date: August 2010
Study Completion Date: September 2011
Primary Completion Date: September 2011 (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 1998 mg per day for those subjects weighing greater than 60kg and 1332 mg per day for those less weighing less than 60kg.
Drug: Acamprosate
Other Name: Campral
No Intervention: Autism Spectrum Disorder
This baseline comparison group will participated in only the psychophysiological and biomarker portion of subject characterization.

  Eligibility

Ages Eligible for Study:   5 Years to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  • Male and female outpatients between the ages of 5 and 17 years.
  • Confirmed diagnosis of Fragile X Syndrome based upon genetic testing.
  • Stable dosing of all psychotropic medications for at least 2 weeks prior to baseline.
  • Subjects with a stable seizure disorder or history of only childhood febrile seizures will be included.
  • Clinical Global Impression-Severity Score of 4 (Moderately Ill) or greater.
  • Must be in good physical health.
  • Subjects of child bearing age of both genders will be required to utilize birth control as applicable.

Exclusion Criteria:

  • Diagnosis of schizophrenia, another psychotic disorder, bipolar disorder or alcohol or other substance abuse based on Diagnostic and Statistical Manual Fourth Edition-Text Revised (DSM-IV-TR).
  • A significant medical condition such as heart, liver, renal or pulmonary disease or unstable seizure disorder.
  • Females with a positive urine pregnancy test
  • Creatinine clearance of less than 30.
  • Concomitant use of another glutamatergic agent (memantine,riluzole, d-cycloserine, amantadine topiramate, gabapentin, among others.
  • Evidence of hypersensitivity to acamprosate or potentially serious adverse effect.
  • 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
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: NCT01300923

Locations
United States, Indiana
Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children
Indianapolis, Indiana, United States, 46202
Sponsors and Collaborators
Indiana University
Investigators
Principal Investigator: Craig A. Erickson, M.D. Indiana University School of Medicine - Department of Psychiatry
  More Information

Publications:
Responsible Party: Indiana University
ClinicalTrials.gov Identifier: NCT01300923     History of Changes
Other Study ID Numbers: 1003-26
Study First Received: August 25, 2010
Results First Received: July 29, 2015
Last Updated: April 27, 2017

Keywords provided by Indiana University:
Fragile X Syndrome
Acamprosate

Additional relevant MeSH terms:
Fragile X Syndrome
Syndrome
Autism Spectrum Disorder
Disease
Pathologic Processes
Child Development Disorders, Pervasive
Neurodevelopmental Disorders
Mental Disorders
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
Acamprosate
Alcohol Deterrents

ClinicalTrials.gov processed this record on May 24, 2017