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

NCT03515213 | Completed Phase 2 Results DMC FDA Drug

• 1 other identifier: 20163022
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 1

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.

Conditions

Huntington Disease

Outcome Measures

Primary Outcomes (2)

• 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 Outcomes (5)

• Change in Fenofibric Acid Level.

  Baseline to 3 and 6 months.

• Change in Unified Huntington Disease Rating Scale Motor Score.

  Baseline and 6 months

• Change in Montreal Cognitive Assesment Score.

  Baseline compared to 3 and 6 months.

• Changes in Unified Huntington Disease Rating Scale - Behavioral

  Baseline, Month 3 and 6 months

• Change in Functional Assessment Scores

  Baseline compared to 3 and 6 months.

Study Arms (2)

Active

ACTIVE COMPARATOR

Drug: Fenofibrate

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

Fenofibrate DRUG

145mg of fenofibrate

Active

Placebo DRUG

Placebo

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• University of California, Irvine: lead

Study Sites (1)

University of California, Irvine

Irvine, California, 92697, United States

Location

MeSH Terms

Conditions

Huntington Disease

Interventions

Fenofibrate

Condition Hierarchy (Ancestors)

Basal Ganglia Diseases; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Dementia; Chorea; Dyskinesias; Movement Disorders; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Cognition Disorders; Neurocognitive Disorders; Mental Disorders

Intervention Hierarchy (Ancestors)

Fibric Acids; Isobutyrates; Butyrates; Acids, Acyclic; Carboxylic Acids; Organic Chemicals; Phenyl Ethers; Ethers; Benzophenones; Benzene Derivatives; Hydrocarbons, Aromatic; Hydrocarbons, Cyclic; Hydrocarbons; Phenols; Ketones

Results Point of Contact

• Title: Leslie Thompson
• Organization: University of California, Irvine

lmthomps@hs.uci.edu

9498243990

Study Officials

• Neal Hermanowicz, MD

  University of California, Irvine

  PRINCIPAL INVESTIGATOR

• Leslie Thompson, PhD

  University of California, Irvine

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: No

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: TRIPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 2, 2017
• First Posted: May 3, 2018
• Study Start: April 27, 2017
• Primary Completion: August 31, 2021
• Study Completion: August 31, 2021
• Last Updated: September 28, 2023
• Results First Posted: September 28, 2023

Record last verified: 2023-09

Data Sharing

• IPD Sharing: Will not share

Locations

US (1)