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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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Active | Active Comparator |
| |
| Placebo | Placebo Comparator |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Fenofibrate | Drug | 145mg of fenofibrate |
| |
| Placebo |
| Measure | Description | Time Frame |
|---|---|---|
| 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. |
| Measure | Description | Time Frame |
|---|---|---|
| Change in Fenofibric Acid Level. | Change in Fenofibric acid abundance from Baseline to 3 and 6 months. | Baseline to 3 and 6 months. |
| Change in Unified Huntington Disease Rating Scale Motor Score. |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Neal Hermanowicz, MD | University of California, Irvine | Principal Investigator |
| Leslie Thompson, PhD | University of California, Irvine | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of California, Irvine | Irvine | California | 92697 | United States |
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| ID | Title | Description |
|---|---|---|
| FG000 | Active | Fenofibrate: 145mg of fenofibrate |
| FG001 | Placebo | Placebo: Placebo |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
Ten participants baseline assessments analyzed.
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| ID | Title | Description |
|---|---|---|
| BG000 | Active | Fenofibrate: 145mg of fenofibrate |
| BG001 | Placebo | Placebo: Placebo |
| BG002 |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Change in PGC-1alpha RNA Expression | Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6 | Fenofibrate vs placebo | Posted | Mean | Standard Deviation | Log 2 fold change | Baseline compared to 3 and 6 months. |
|
|
Adverse events data were collected over 6 months.
All-Cause Mortality, Serious, or Other (non-serious) Adverse Events were monitored/assessed, but no adverse events were observed over 6 months.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Active | Fenofibrate: 145mg of fenofibrate | 0 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Leslie Thompson | University of California, Irvine | 9498243990 | lmthomps@hs.uci.edu |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot_SAP | Yes | Yes | No | Study Protocol and Statistical Analysis Plan | Aug 24, 2018 | Feb 1, 2023 | Prot_SAP_000.pdf |
| ICF | No | No | Yes | Informed Consent Form | Aug 24, 2018 | Feb 1, 2023 | ICF_001.pdf |
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| ID | Term |
|---|---|
| D006816 | Huntington Disease |
| ID | Term |
|---|---|
| D001480 | Basal Ganglia Diseases |
| D001927 | Brain Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
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| ID | Term |
|---|---|
| D011345 | Fenofibrate |
| ID | Term |
|---|---|
| D058607 | Fibric Acids |
| D058610 | Isobutyrates |
| D002087 | Butyrates |
| D000144 | Acids, Acyclic |
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| Drug |
Placebo |
|
Unified Huntington Disease Rating Scale (UHDRS) Motor (total score range 0-124 where higher score = greater impairment)
| Baseline and 6 months |
| Change in Montreal Cognitive Assesment Score. | Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment) | 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) | 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) | Baseline compared to 3 and 6 months. |
| Total |
Total of all reporting groups |
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Ethnicity (NIH/OMB) | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| UHDRS TFC | United Huntington Disease Rating Scale, Total Functional Capacity, greater than or equal to 7 was the inclusion criteria and is a scale representing functional capacity. A higher score represents worsening and lower score represents better functional capacity. | Mean | Standard Deviation | units on a scale |
|
| Participants |
|
|
| Primary | Change in PGC-1alpha Protein Abundance. | Mean change in PGC-1alpha protein abundance. | Posted | Mean | Standard Deviation | Log 2 fold change | Baseline compared to 3 and 6 months. |
|
|
|
| Secondary | Change in Fenofibric Acid Level. | Change in Fenofibric acid abundance from Baseline to 3 and 6 months. | Fenofibrate vs placebo | Posted | Mean | Standard Deviation | Ng/ml | Baseline to 3 and 6 months. |
|
|
|
| Secondary | 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) | Fenofibrate vs placebo. Change in motor scores from baseline to month 6. | Posted | Mean | Standard Deviation | score on a scale | Baseline and 6 months |
|
|
|
| Secondary | Change in Montreal Cognitive Assesment Score. | Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment) | Changes in cognition scores from baseline to month 6. | Posted | Mean | Standard Deviation | score on a scale | Baseline compared to 3 and 6 months. |
|
|
|
| Secondary | 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) | Changes in independence assessment scores from baseline, month 3 to month 6. | Posted | Mean | Standard Deviation | score on a scale | Baseline, Month 3 and 6 months |
|
|
|
| Secondary | 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) | Change in functional assessment scores from baseline, month 3 to month 6. | Posted | Mean | Standard Deviation | score on a scale | Baseline compared to 3 and 6 months. |
|
|
|
| 9 |
| 0 |
| 9 |
| 0 |
| 9 |
| EG001 | Placebo | Placebo: Placebo | 0 | 1 | 0 | 1 | 0 | 1 |
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| D003704 | Dementia |
| D002819 | Chorea |
| D020820 | Dyskinesias |
| D009069 | Movement Disorders |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D003072 | Cognition Disorders |
| D019965 | Neurocognitive Disorders |
| D001523 | Mental Disorders |
| D002264 |
| Carboxylic Acids |
| D009930 | Organic Chemicals |
| D010647 | Phenyl Ethers |
| D004987 | Ethers |
| D001577 | Benzophenones |
| D001555 | Benzene Derivatives |
| D006841 | Hydrocarbons, Aromatic |
| D006844 | Hydrocarbons, Cyclic |
| D006838 | Hydrocarbons |
| D010636 | Phenols |
| D007659 | Ketones |
| Month 6 |
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| Month 6 |
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| Month 6 |
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| Month 6 |
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