Phase 2
Completed N=6
Treatment of Fragile-X Associated Tremor/Ataxia Syndrome (FXTAS) With Allopregnanolone
Fragile X-associated Tremor/Ataxia Syndrome
Source: ClinicalTrials.gov NCT02603926 ↗
Enrolled (actual)
6
Serious AEs
0.0%
Results posted
Dec 2018
Primary outcomePrimary: California Verbal Learning Test II (CVLT2) Trial 1-5 Free Recall Total Raw Score — 34.0; 33.5 score on a scale
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).
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 | — |
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
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. Informational only — not medical advice.