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

University of California, Irvine10 enrolled

Overview

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 Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

TRIPLE

Enrollment

Conditions

Huntington Disease

Interventions

FenofibrateDRUG

145mg of fenofibrate

PlaceboDRUG

Placebo

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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.

Locations (1)

University of California, Irvine

Irvine, California, United States

Outcomes

Primary Outcomes

Change in PGC-1alpha RNA Expression

Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6

Time frame: Baseline compared to 3 and 6 months.

Change in PGC-1alpha Protein Abundance.

Mean change in PGC-1alpha protein abundance.

Time frame: Baseline compared to 3 and 6 months.

Secondary Outcomes

Change in Fenofibric Acid Level.

Change in Fenofibric acid abundance from Baseline to 3 and 6 months.

Time frame: 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)

Time frame: Baseline and 6 months

Change in Montreal Cognitive Assesment Score.

Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment)

Time frame: 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)

Time frame: 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)

Data from ClinicalTrials.gov

This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.