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Phase 2 N=10 Randomized Triple-blind Treatment

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

Huntington Disease

Enrolled (actual)
10
Serious AEs
0.0%
Results posted
Sep 2023
Primary outcome: Primary: Change in PGC-1alpha RNA Expression — -0.02190716; 0.3160562; 0.07414876; 0.4800263 Log 2 fold change

Study Design & Population

Study type
Interventional
Phase
Phase 2
Interventions
Fenofibrate (Drug); Placebo (Drug)
Age
Adult, Older Adult · 18+ yrs
Sex
All
Sponsor
University of California, Irvine
Primary completion
Aug 2021

Outcome Measures

OutcomeResultp-value
PRIMARY
Change in PGC-1alpha RNA Expression
-0.02190716; 0.3160562; 0.07414876; 0.4800263
PRIMARY
Change in PGC-1alpha Protein Abundance.
0.2609123; -0.02029792; 0.02375246; 0.3185422
SECONDARY
Change in Fenofibric Acid Level.
64; 5; 9037; 10; 10905; 19
SECONDARY
Change in Unified Huntington Disease Rating Scale Motor Score.
19.56; 34; 21.22; 27
SECONDARY
Change in Montreal Cognitive Assesment Score.
24.222; 18; 25.778; 16; 25.667; 22
SECONDARY
Changes in Unified Huntington Disease Rating Scale - Behavioral
9.778; 8; 8.333; 9; 8.444; 8
SECONDARY
Change in Functional Assessment Scores
3.222; 3; 3.571; 3; 3.571; 3

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.

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.
View full record on ClinicalTrials.gov →

Data sourced from ClinicalTrials.gov (NCT03515213). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.

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