Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease

Study Overview
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Brief Summary
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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 triglycerides (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.

Official Title
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A Phase IIa, Randomized, Double-blind, Placebo-controlled Study of the Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease

Conditions
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Huntington Disease

Intervention / Treatment
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* Drug: Fenofibrate
* Drug: Placebo


Participation Criteria
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Eligibility Criteria
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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.

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Triple


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Active Comparator: Active<br> | Drug: Fenofibrate<br>* 145mg of fenofibrate<br>|
| Placebo Comparator: Placebo<br> | Drug: Placebo<br>* Placebo<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in PGC-1alpha RNA Expression | Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6 | Baseline compared to 3 and 6 months. | 
| Change in PGC-1alpha Protein Abundance. | Mean change in PGC-1alpha protein abundance. | Baseline compared to 3 and 6 months. | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Change in Fenofibric Acid Level. | Change in Fenofibric acid abundance from Baseline to 3 and 6 months. | Baseline to 3 and 6 months. | 
| Change in Unified Huntington Disease Rating Scale Motor Score. | Unified Huntington Disease Rating Scale (UHDRS) Motor (total score range 0-124 where higher score = greater impairment) | Baseline and 6 months | 
| Change in Montreal Cognitive Assesment Score. | Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment) | Baseline compared to 3 and 6 months. | 
| Changes in Unified Huntington Disease Rating Scale - Behavioral | Unified Huntington Disease Rating Scale (UHDRS) Behavioral Scale (total score range 0-100 where 100 = 100% independent) | Baseline, Month 3 and 6 months | 
| Change in Functional Assessment Scores | 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) | Baseline compared to 3 and 6 months. |