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

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT02603926
• Date of registration: 05/11/2015
• Prospective Registration: No
• Primary sponsor: Randi J. Hagerman, MD
• Public title: Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
• Scientific title: Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
• Date of first enrolment: October 2015
• Target sample size: 6
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02603926
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Randi J Hagerman, MD
• Affiliation: UC Davis MIND Institute

Key inclusion & exclusion 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



Age minimum: 50 Years
Age maximum: 85 Years
Gender: All

Health Condition(s) or Problem(s) studied

Fragile X-associated Tremor/Ataxia Syndrome

Intervention(s)

Drug: Allopregnanolone

Primary Outcome(s)

California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score [Time Frame: Baseline/pre-treatment and 14 weeks/post-treatment]

Secondary Outcome(s)

Behavioral Dyscontrol Scale - 2 (BDS-2) Total Score [Time Frame: Baseline/pre-treatment and 14 weeks/post-treatment]

Hippocampal Volume, as Measured by Structural MRI [Time Frame: Baseline/pre-treatment and 14 weeks/post-treatment]

CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI) [Time Frame: Baseline/pre-treatment and 14 weeks/post-treatment]

Secondary ID(s)

720668

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 06/12/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02603926