Huntington DiseasePhase 2NCT03515213
Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease
A Phase 2 study testing Fenofibrate in people with Huntington Disease, run by University of California, Irvine at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Huntington Disease
- What is being tested?
- Fenofibrate
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 10 participants
- Where
- 1 siteUnited States
- Study dates
- 27 Apr 2017to 31 Aug 2021
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
University of California, Irvine
Irvine, California, 92697
About this study
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
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
- Being tested
- Fenofibrate, Placebo
- Medical subject headings
- Huntington Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
FenofibrateDrug
145mg of fenofibrate
PlaceboDrug
Placebo
| Study group | Role | What they receive |
|---|---|---|
| Active | Active comparator | Not described Receives: Drug: Fenofibrate |
| Placebo | Placebo comparator | Not described Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in PGC-1alpha RNA Expression
Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6
Measured: Baseline compared to 3 and 6 months.
Change in PGC-1alpha Protein Abundance.
Mean change in PGC-1alpha protein abundance.
Measured: Baseline compared to 3 and 6 months.
Secondary outcomes (5)
Change in Fenofibric Acid Level.
Change in Fenofibric acid abundance from Baseline to 3 and 6 months.
Measured: 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)
Measured: Baseline and 6 months
Change in Montreal Cognitive Assesment Score.
Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment)
Measured: 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)
Measured: 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)
Measured: Baseline compared to 3 and 6 months.
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 May 2018
- Study start27 Apr 2017
- Primary completion31 Aug 2021
- Study completion31 Aug 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Huntington DiseaseStudies of the same condition, open and under way.
- More trials run by University of California, IrvineThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind
- Who is masked
- Participant, Care provider, Investigator
- Enrolment
- 10 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of California, Irvine
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Leslie Thompson, University of California, Irvine
- Investigators
- Neal Hermanowicz, MD, Principal investigator, University of California, IrvineLeslie Thompson, PhD, Study director, University of California, Irvine
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03515213
- Sponsor's protocol ID
- 20163022
- Status last verified
- Sep 2023
- First submitted
- 2 Aug 2017
- Last updated
- 28 Sep 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 28 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT03515213
- Acronym / sponsor ID
- 20163022
- Conditions
- Huntington Disease
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 10 participants (actual)
- Lead sponsor
- University of California, IrvineUniversity, hospital or other
- Study start
- 27 Apr 2017 (actual)
- Primary completion
- 31 Aug 2021 (actual)
- Study completion
- 31 Aug 2021 (actual)
- First posted
- 3 May 2018 (actual)
- Results
- Posted 28 Sep 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Triple blind (participant, care provider, investigator)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Active | Active comparator | Drug: Fenofibrate |
| Placebo | Placebo comparator | Drug: Placebo |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fenofibrate145mg of fenofibrate | Drug | Active |
| PlaceboPlacebo | Drug | Placebo |
Primary outcome
Change in PGC-1alpha RNA Expression
Time frame: Baseline compared to 3 and 6 months.
Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6
Change in PGC-1alpha Protein Abundance.
Time frame: Baseline compared to 3 and 6 months.
Mean change in PGC-1alpha protein abundance.
5 secondary outcomes
Change in Fenofibric Acid Level.
Time frame: Baseline to 3 and 6 months.
Change in Unified Huntington Disease Rating Scale Motor Score.
Time frame: Baseline and 6 months
Change in Montreal Cognitive Assesment Score.
Time frame: Baseline compared to 3 and 6 months.
Changes in Unified Huntington Disease Rating Scale - Behavioral
Time frame: Baseline, Month 3 and 6 months
Change in Functional Assessment Scores
Time frame: Baseline compared to 3 and 6 months.
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 28 Sep 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Individual participant dataSharing planned: No
Related trials for Huntington Disease
RecruitingPhase 2/3
Study to Evaluate the Pharmacodynamics, Safety and Efficacy of SKY-0515 in Participants With Huntington's Disease
Huntington Disease
RecruitingPhase 3
Pridopidine Phase 3 Study in Huntington's Disease
Huntington Disease
Not yet recruiting
Creatine and Beta-Hydroxy-Beta-Methylbutyrate (HMB) Supplementation Plus Home-Based Exercise for Sarcopenia in Huntington's Disease
Huntington Disease, Sarcopenia
Recruiting
Feasibility and Acceptability of an LLM-based Chatbot for Family Caregivers: Evaluation Study
Caregivers
RecruitingObservational study
Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders
Invasive PreNatal Diagnosis in a Context of Family History of Single-gene Disorders, Including, Sickle Cell Disease
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Neal Hermanowicz, MD principal investigator
University of California, Irvine
Leslie Thompson, PhD study director
University of California, Irvine
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fenofibrate in people with Huntington Disease, run by University of California, Irvine at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 28 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 28 Sep 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in PGC-1alpha RNA Expression (time frame: Baseline compared to 3 and 6 months.). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 28 Sep 2023.