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

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

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.

Study Overview

• Status: Completed
• Conditions: Huntington Disease
• Intervention / Treatment: Drug: Placebo; Drug: Fenofibrate

• Study Type: Interventional
• Enrollment (Actual): 10
• Phase: Phase 2

Contacts and Locations

Study Locations

• United States
  • California
    • Irvine, California, United States, 92697
      • University of California, Irvine

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 16 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Triple

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Placebo Comparator: Placebo	Drug: Placebo; Placebo
Active Comparator: Active	Drug: Fenofibrate; 145mg of fenofibrate

What is the study measuring?

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.

Collaborators and Investigators

• Sponsor: University of California, Irvine
• Investigators: Principal Investigator: Neal Hermanowicz, MD, University of California, Irvine; Study Director: Leslie Thompson, PhD, University of California, Irvine

Study record dates

Study Major Dates

• Study Start (Actual): April 27, 2017
• Primary Completion (Actual): August 31, 2021
• Study Completion (Actual): August 31, 2021

Study Registration Dates

• First Submitted: August 2, 2017
• First Submitted That Met QC Criteria: May 1, 2018
• First Posted (Actual): May 3, 2018

Study Record Updates

• Last Update Posted (Actual): September 28, 2023
• Last Update Submitted That Met QC Criteria: September 3, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Neurocognitive Disorders; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Dyskinesias; Heredodegenerative Disorders, Nervous System; Dementia; Cognition Disorders; Chorea; Huntington Disease; Molecular Mechanisms of Pharmacological Action; Antimetabolites; Hypolipidemic Agents; Lipid Regulating Agents; Fenofibrate
• Other Study ID Numbers: 20163022

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No
• product manufactured in and exported from the U.S.: No