Acamprosate in Youth With Fragile X Syndrome

NCT01300923 | Completed Phase 2 Results DMC

• 1 other identifier: 1003-26
• Study Type: interventional
• Target: 14
• Locations: 1 country
• Sites: 1

Brief Summary

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.

Conditions

Fragile X Syndrome; Autism Spectrum Disorders

Keywords

Fragile X Syndrome; Acamprosate

Outcome Measures

Primary Outcomes (1)

• Clinical Global Impression- Severity Scale (CGI-S)

  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.

  Week 10

Secondary Outcomes (7)

• The Aberrant Behavior Checklist (ABC)

  Week 10

• Social Responsiveness Scale

  Week 10

• Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD

  Week 10

• ADHD Rating Scale 4th Edition

  Week 10

• Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain

  Week 10

Study Arms (2)

Acamprosate

ACTIVE COMPARATOR

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

Autism Spectrum Disorder

NO INTERVENTION

This baseline comparison group will participated in only the psychophysiological and biomarker portion of subject characterization.

Interventions

Acamprosate DRUG

Also known as: Campral

Acamprosate

Eligibility Criteria

• Age: 5 Years - 17 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Indiana University: 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, Wink LK, Ray B, Early MC, Stiegelmeyer E, Mathieu-Frasier L, Patrick V, Lahiri DK, McDougle CJ. Impact of acamprosate on behavior and brain-derived neurotrophic factor: an open-label study in youth with fragile X syndrome. Psychopharmacology (Berl). 2013 Jul;228(1):75-84. doi: 10.1007/s00213-013-3022-z. Epub 2013 Feb 24.

  PMID: 23436129 BACKGROUND

MeSH Terms

Conditions

Fragile X Syndrome; Autism Spectrum Disorder

Interventions

Acamprosate

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; Child Development Disorders, Pervasive; Neurodevelopmental Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Taurine; Alkanesulfonic Acids; Alkanes; Hydrocarbons, Acyclic; Hydrocarbons; Organic Chemicals; Sulfonic Acids; Sulfur Acids; Sulfur Compounds

Limitations and Caveats

Psychophysiological measures proved non-feasible as subjects could not tolerate the procedures. The paradigms were tried on 2 subjects in the ADS arm, as data collected was not usable. Subsequently, neurobiological characterization was halted.

Results Point of Contact

• Title: Craig Erickson
• Organization: Cincinnati Childrens Hospital

craig.erickson@cchmc.org

5136366265

Study Officials

• Craig A. Erickson, M.D.

  Indiana University School of Medicine - Department of Psychiatry

  PRINCIPAL INVESTIGATOR

Publication Agreements

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

Study Design

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

Study Record Dates

• First Submitted: August 25, 2010
• First Posted: February 23, 2011
• Study Start: August 1, 2010
• Primary Completion: September 1, 2011
• Study Completion: September 1, 2011
• Last Updated: July 30, 2019
• Results First Posted: April 28, 2017

Record last verified: 2019-07

Locations

US (1)