Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease
Study Details
Study Description
Brief Summary
The purpose of this research study is to study the safety and efficacy of fenofibrate, an FDA-approved drug for high cholesterol and/or elevated triglyerides (fats), as a treatment for Huntington's disease (HD). Subjects who meet the entry criteria will be randomized (3:1) to either 145mg of fenofibrate or placebo.
| Condition or Disease | Intervention/Treatment | Phase |
|---|---|---|
|
Phase 2 |
Study Design
Arms and Interventions
| Arm | Intervention/Treatment |
|---|---|
| Active Comparator: Active
|
Drug: Fenofibrate
145mg of fenofibrate
|
| Placebo Comparator: Placebo
|
Drug: Placebo
Placebo
|
Outcome Measures
Primary Outcome Measures
- Change in PGC-1alpha from Baseline to Month 6 [Baseline, 1 month, 2 months, 3 months, 4 months, 5 months and 6 months]
Change in PGC-1alpha from Baseline to Month 6
Secondary Outcome Measures
- Change in Fenofibrate level from Baseline to Month 6 [Baseline, 1 month, 2 months, 3 months, 4 months, 5 months and 6 months]
Change in Fenofibrate level from Baseline to Month 6
- Change in Motor Scores from Baseline to Month 6 [Baseline, 3 months, and 6 months]
Unified Huntington Disease Rating Scale (UHDRS) Motor (total score range 0-124 where higher score = greater impairment)
- Change in Cognition Scores from Baseline to Month 6 [Baseline, 3 months, and 6 months]
Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment), Stroop Word (cognitive interference measure, calculated interference score where higher = better performance), and Benton Judgement of Line Orientation (test of spatial thinking, total score range 0-30, higher score = better performance)
- Changes in Independence Assessment Scores from Baseline to Month 6 [Baseline, 3 months, and 6 months]
Unified Huntington Disease Rating Scale (UHDRS) Independence Scale (total score range 0-100 where 100 = 100% independent)
- Change in Functional Assessment Scores from Baseline to Month 6 [Baseline, 3 months, and 6 months]
Clinical Global Impression (CGI-I); (total score range 1-7 where higher score = greater impairment), and Unified Huntington Disease Rating Scale (UHDRS) Total Functional Capacity Assessment ( total score range 0-13, higher score = less impairment)
Eligibility Criteria
Criteria
Inclusion Criteria:
-
An adult of either sex, ages 25-85 inclusive,
-
Have proficiency with written and spoken English and corrected vision or hearing to complete the cognitive testing,
-
Are able to give informed consent,
-
Have good overall health status with no known problems anticipated over the course of the trial,
-
Have a diagnosis of HD supported by positive gene test within the past 6 months.
Exclusion Criteria:
-
Other major neurological disease [e.g., multiple sclerosis, parkinson's disease, cortical stroke, etc]
-
Clinically significant hepatic or renal disease,
-
Current or recent (< 1 month) use of dopamine blocking agents such as tetrabenazine, anticonvulsants, neuroleptics, HAART, antiemetics, and antipsychotics for any reason,
-
Current use of Warfarin (Coumadin). Enrollment in another investigational drug study within the prior three months.
Contacts and Locations
Locations
| Site | City | State | Country | Postal Code | |
|---|---|---|---|---|---|
| 1 | University of California, Irvine | Irvine | California | United States | 92697 |
Sponsors and Collaborators
- University of California, Irvine
Investigators
- Principal Investigator: Neal Hermanowicz, MD, University of California, Irvine
- Study Director: Leslie Thompson, PhD, University of California, Irvine
Study Documents (Full-Text)
None provided.More Information
Publications
None provided.- 2016-3022