Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone

• Status: Completed
• Trial end date: 2017-12-01
• Target enrollment: 0
• Participant gender: All

Summary

The purpose of this study is to examine the safety and efficacy of Allopregnanolone as a possible treatment for symptoms of Fragile X-associated Tremor/Ataxia Syndrome (FXTAS).

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Randi J. Hagerman, MD; University of California, Davis
• Treatments: Brexanolone; Pregnanolone

Criteria

Inclusion Criteria

• Fragile X premutation carrier status (55 to 200 CGG repeats in FMR1),

• Diagnosis of FXTAS including an intention tremor and/or ataxia and/or deficits on the BDS-2 demonstrating executive function deficits.

Exclusion Criteria

• other genetic problems in addition to the premutation

• a history of significant brain trauma

• significant substance abuse

• inability to follow the protocol

• liver or kidney disease

• heart failure

• active cancer

• other serious systemic disease

• current use of phenytoin