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

Phase 2

Completed

• Conditions: Huntington Disease
• Interventions: Drug: Placebo; Drug: Fenofibrate

• Registration Number: NCT03515213
• Lead Sponsor: University of California, Irvine

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 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.

Detailed Description

Not available

Study Timeline

• Study Start: 2017-04-27
• Study First Submitted: 2017-08-02
• Study First Submitted QC: 2018-05-01
• Study First Posted: 2018-05-03
• Primary Completion: 2021-08-31
• Study Completion: 2021-08-31
• Results First Submitted: 2023-02-01
• Status Verified: 2023-09
• Results First Submitted QC: 2023-09-03
• Last Update Submitted: 2023-09-03
• Results First Posted: 2023-09-28
• Last Update Posted: 2023-09-28

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 10

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo	Placebo	-
Active	Fenofibrate	-

Primary Outcome Measures

Name	Time	Method
Change in PGC-1alpha RNA Expression	Baseline compared to 3 and 6 months.	Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6
Change in PGC-1alpha Protein Abundance.	Baseline compared to 3 and 6 months.	Mean change in PGC-1alpha protein abundance.

Secondary Outcome Measures

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

Trial Locations

Locations (1)

University of California, Irvine; 🇺🇸 Irvine, California, United States