Clinical trial NCT01300923: Acamprosate in Youth With Fragile X Syndrome
Official title: Pilot Study of Acamprosate in Youth With Fragile X Syndrome
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Fragile X Syndrome
Autism Spectrum Disorders
Keywords: Fragile X Syndrome
Acamprosate
Lead sponsor: Indiana University
Sponsor class: OTHER
First posted: 2011-02-23
Enrollment: 14
Sex: ALL
Minimum age (years): 5
Maximum age (years): 17
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.


Eligibility 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.

Primary outcomes:
measure: Clinical Global Impression- Severity Scale (CGI-S)
timeFrame: Week 10
Secondary outcomes:
measure: The Aberrant Behavior Checklist (ABC)
timeFrame: Week 10
measure: Social Responsiveness Scale
timeFrame: Week 10
measure: Children's Yale-Brown Obsessive Compulsive Scale Modified for PDD
timeFrame: Week 10
measure: ADHD Rating Scale 4th Edition
timeFrame: Week 10
measure: Vineland Adaptive Behavior Scales-II (VABS-II) Communication Domain
timeFrame: Week 10
measure: Peabody Picture Vocabulary
timeFrame: Week 10
measure: Brain-derived Neurotrophic Factor (BDNF)
timeFrame: Screen and Week 10
Other outcomes:
Locations:
city: Indianapolis
country: United States
facility: Riley Child and Adolescent Psychiatry Clinic - Riley Hospital for Children
state: Indiana
Study officials:
affiliation: Indiana University School of Medicine - Department of Psychiatry
name: Craig A. Erickson, M.D.
role: PRINCIPAL_INVESTIGATOR
References:
citation: 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.