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

Fragile X-associated Tremor/Ataxia Syndrome Clinical Trial

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

Status Completed

Clinical Trial 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).

Clinical Trial Description

This study includes a screening visit with several assessments, followed by an open-label medication trial of Allopregnanolone for 12 weeks and an end-point evaluation to assess for changes. Assessments include blood draws for genetic and safety laboratory testing, neurological and physical exam and medical history, cognitive testing, and motor testing.

Study record was updated in October 2018 to include adverse events and outcome measure reporting. Study record was updated in November 2018 in response to requests to (1) specify time frame of reported outcome measures, (2) clarify that the RASS was a safety monitoring tool, not a prespecified outcome measure, and as such will not be reported as an outcome measure, and (3) upload a version of the study protocol and statistical analysis plan with the required title page and statistical analysis plan information. ;

Related Conditions & MeSH terms

• Ataxia
• Cerebellar Ataxia
• Fragile X Syndrome
• Fragile X-associated Tremor/Ataxia Syndrome
• Syndrome
• Tremor

• NCT number: NCT02603926
• Study type: Interventional
• Source: University of California, Davis
• Status: Completed
• Phase: Phase 2
• Start date: October 2015
• Completion date: December 2017