Acamprosate in Youth With Fragile X Syndrome

Indiana University

Status and phase: Completed; Phase 2

Conditions: Fragile X Syndrome; Autism Spectrum Disorders

Treatments: Drug: Acamprosate

Study type: Interventional

Funder types: Other

Identifiers

NCT01300923

1003-26

Details and patient eligibility

About

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.

Enrollment: 14 patients

Sex: All

Ages: 5 to 17 years old

Volunteers: No Healthy Volunteers

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.

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Single Group Assignment

Masking: None (Open label)

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14 participants in 2 patient groups

Acamprosate

Active Comparator group

Description:

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.

Treatment:

Drug: Acamprosate

Autism Spectrum Disorder

No Intervention group

Description:

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