Phase 2 N=6 Treatment

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

Fragile X-associated Tremor/Ataxia Syndrome

Bottom Line

View on ClinicalTrials.gov: NCT02603926 ↗

Enrolled (actual)

Serious AEs

0.0%

Results posted

Dec 2018

Primary outcome: Primary: California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score — 34.0; 33.5 score on a scale

Study Design & Population

Study type: Interventional
Phase: Phase 2
Interventions: Allopregnanolone (Drug)
Age: Adult, Older Adult · 50+ yrs
Sex: All
Sponsor: Randi J. Hagerman, MD
Primary completion: Dec 2016

Outcome Measures

Outcome	Result	p-value
PRIMARY California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score	34.0; 33.5	—
SECONDARY Behavioral Dyscontrol Scale - 2 (BDS-2) Total Score	12.7; 15.3	—
SECONDARY CATSYS Dot-to-Dot Tremor Intensity (CATSYS DTD TI)	0.87; 0.74; 0.79; 0.65	—
SECONDARY Hippocampal Volume, as Measured by Structural MRI	5.87; 5.84	—

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).

Eligibility 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

View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT02603926). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.