Study of Acamprosate in Fragile x Syndrome

Cincinnati Children's Hospital Medical Center

Status and phase: Completed; Phase 1

Conditions: Fragile X Syndrome

Treatments: Drug: Placebo; Drug: acamprosate

Study type: Interventional

Funder types: Other

Identifiers

NCT01911455

CIN001- Acamprosate in FX

Details and patient eligibility

About

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).

Full description

Each subject with FXS will receive 10 weeks of blinded treatment with acamprosate or matching placebo. After completion of the double-blind phase, all subjects will have an opportunity to receive acamprosate as part of the study procedures for 16 weeks.

Enrollment: 46 patients

Sex: All

Ages: 5 to 23 years old

Volunteers: Accepts Healthy Volunteers

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

Trial design

Primary purpose: Basic Science

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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46 participants in 2 patient groups, including a placebo group

acamprosate

Active Comparator group

Description:

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.

Treatment:

Drug: acamprosate

Placebo

Placebo Comparator group

Description:

Placebo will be prescribed with the same frequency and duration as the acamprosate group.

Treatment:

Drug: Placebo