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

Primary Purpose

Huntington Disease

Status
Completed
Phase
Phase 2
Locations
United States
Study Type
Interventional
Intervention
Fenofibrate
Placebo
Sponsored by
University of California, Irvine
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Huntington Disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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.

Sites / Locations

  • University of California, Irvine

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Placebo Comparator

Arm Label

Active

Placebo

Arm Description

Outcomes

Primary Outcome Measures

Change in PGC-1alpha RNA Expression
Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6
Change in PGC-1alpha Protein Abundance.
Mean change in PGC-1alpha protein abundance.

Secondary Outcome Measures

Change in Fenofibric Acid Level.
Change in Fenofibric acid abundance from 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)
Change in Montreal Cognitive Assesment Score.
Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment)
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)
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)

Full Information

First Posted
August 2, 2017
Last Updated
September 3, 2023
Sponsor
University of California, Irvine
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1. Study Identification

Unique Protocol Identification Number
NCT03515213
Brief Title
Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease
Official Title
A Phase IIa, Randomized, Double-blind, Placebo-controlled Study of the Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease
Study Type
Interventional

2. Study Status

Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
April 27, 2017 (Actual)
Primary Completion Date
August 31, 2021 (Actual)
Study Completion Date
August 31, 2021 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
University of California, Irvine

4. Oversight

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
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Huntington Disease

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Masking
ParticipantCare ProviderInvestigator
Allocation
Randomized
Enrollment
10 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Active
Arm Type
Active Comparator
Arm Title
Placebo
Arm Type
Placebo Comparator
Intervention Type
Drug
Intervention Name(s)
Fenofibrate
Intervention Description
145mg of fenofibrate
Intervention Type
Drug
Intervention Name(s)
Placebo
Intervention Description
Placebo
Primary Outcome Measure Information:
Title
Change in PGC-1alpha RNA Expression
Description
Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6
Time Frame
Baseline compared to 3 and 6 months.
Title
Change in PGC-1alpha Protein Abundance.
Description
Mean change in PGC-1alpha protein abundance.
Time Frame
Baseline compared to 3 and 6 months.
Secondary Outcome Measure Information:
Title
Change in Fenofibric Acid Level.
Description
Change in Fenofibric acid abundance from Baseline to 3 and 6 months.
Time Frame
Baseline to 3 and 6 months.
Title
Change in Unified Huntington Disease Rating Scale Motor Score.
Description
Unified Huntington Disease Rating Scale (UHDRS) Motor (total score range 0-124 where higher score = greater impairment)
Time Frame
Baseline and 6 months
Title
Change in Montreal Cognitive Assesment Score.
Description
Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment)
Time Frame
Baseline compared to 3 and 6 months.
Title
Changes in Unified Huntington Disease Rating Scale - Behavioral
Description
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
Title
Change in Functional Assessment Scores
Description
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)
Time Frame
Baseline compared to 3 and 6 months.

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Neal Hermanowicz, MD
Organizational Affiliation
University of California, Irvine
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Leslie Thompson, PhD
Organizational Affiliation
University of California, Irvine
Official's Role
Study Director
Facility Information:
Facility Name
University of California, Irvine
City
Irvine
State/Province
California
ZIP/Postal Code
92697
Country
United States

12. IPD Sharing Statement

Plan to Share IPD
No

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

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