Trial Outcomes & Findings for Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease (NCT NCT03515213)
NCT ID: NCT03515213
Last Updated: 2023-09-28
Results Overview
Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Baseline compared to 3 and 6 months.
Results posted on
2023-09-28
Participant Flow
Participant milestones
| Measure |
Active
Fenofibrate: 145mg of fenofibrate
|
Placebo
Placebo: Placebo
|
|---|---|---|
|
Overall Study
STARTED
|
9
|
1
|
|
Overall Study
COMPLETED
|
9
|
1
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Safety and Efficacy of Fenofibrate as a Treatment for Huntington's Disease
Baseline characteristics by cohort
| Measure |
Active
n=9 Participants
Fenofibrate: 145mg of fenofibrate
|
Placebo
n=1 Participants
Placebo: Placebo
|
Total
n=10 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Categorical
<=18 years
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Age, Categorical
Between 18 and 65 years
|
8 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
9 Participants
n=5 Participants
|
|
Age, Categorical
>=65 years
|
1 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
1 Participants
n=5 Participants
|
|
Age, Continuous
|
54.8 years
STANDARD_DEVIATION 9.9 • n=5 Participants
|
49.9 years
n=7 Participants
|
54.8 years
STANDARD_DEVIATION 9.9 • n=5 Participants
|
|
Sex: Female, Male
Female
|
5 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
4 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
5 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
9 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
9 Participants
n=5 Participants
|
1 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Region of Enrollment
United States
|
9 participants
n=5 Participants
|
1 participants
n=7 Participants
|
10 participants
n=5 Participants
|
|
UHDRS TFC
|
9.78 units on a scale
STANDARD_DEVIATION 2.44 • n=5 Participants
|
8 units on a scale
STANDARD_DEVIATION 0 • n=7 Participants
|
9.6 units on a scale
STANDARD_DEVIATION 2.37 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline compared to 3 and 6 months.Population: Fenofibrate vs placebo
Change in PGC-1alpha RNA expression from Baseline, Month 3 and Month 6
Outcome measures
| Measure |
Active
n=9 Participants
Fenofibrate: 145mg of fenofibrate
|
Placebo
n=1 Participants
Placebo: Placebo
|
|---|---|---|
|
Change in PGC-1alpha RNA Expression
Month 3
|
-0.02190716 Log 2 fold change
Standard Deviation 0.9017336
|
0.3160562 Log 2 fold change
|
|
Change in PGC-1alpha RNA Expression
Month 6
|
0.07414876 Log 2 fold change
Standard Deviation 0.4723322
|
0.4800263 Log 2 fold change
|
PRIMARY outcome
Timeframe: Baseline compared to 3 and 6 months.Mean change in PGC-1alpha protein abundance.
Outcome measures
| Measure |
Active
n=9 Participants
Fenofibrate: 145mg of fenofibrate
|
Placebo
n=1 Participants
Placebo: Placebo
|
|---|---|---|
|
Change in PGC-1alpha Protein Abundance.
Month 3
|
0.2609123 Log 2 fold change
Standard Deviation 1.031904
|
-0.02029792 Log 2 fold change
|
|
Change in PGC-1alpha Protein Abundance.
Month 6
|
0.02375246 Log 2 fold change
Standard Deviation 0.9096516
|
0.3185422 Log 2 fold change
|
SECONDARY outcome
Timeframe: Baseline to 3 and 6 months.Population: Fenofibrate vs placebo
Change in Fenofibric acid abundance from Baseline to 3 and 6 months.
Outcome measures
| Measure |
Active
n=4 Participants
Fenofibrate: 145mg of fenofibrate
|
Placebo
n=1 Participants
Placebo: Placebo
|
|---|---|---|
|
Change in Fenofibric Acid Level.
Baseline
|
64 Ng/ml
Standard Deviation 117.48
|
5 Ng/ml
Standard Deviation 0
|
|
Change in Fenofibric Acid Level.
Month 3
|
9037 Ng/ml
Standard Deviation 7114.1
|
10 Ng/ml
Standard Deviation 0
|
|
Change in Fenofibric Acid Level.
Month 6
|
10905 Ng/ml
Standard Deviation 5900.77
|
19 Ng/ml
Standard Deviation 0
|
SECONDARY outcome
Timeframe: Baseline and 6 monthsPopulation: Fenofibrate vs placebo. Change in motor scores from baseline to month 6.
Unified Huntington Disease Rating Scale (UHDRS) Motor (total score range 0-124 where higher score = greater impairment)
Outcome measures
| Measure |
Active
n=9 Participants
Fenofibrate: 145mg of fenofibrate
|
Placebo
n=1 Participants
Placebo: Placebo
|
|---|---|---|
|
Change in Unified Huntington Disease Rating Scale Motor Score.
Baseline
|
19.56 score on a scale
Standard Deviation 11.87
|
34 score on a scale
Standard Deviation 0
|
|
Change in Unified Huntington Disease Rating Scale Motor Score.
Month 6
|
21.22 score on a scale
Standard Deviation 17.22
|
27 score on a scale
Standard Deviation 0
|
SECONDARY outcome
Timeframe: Baseline compared to 3 and 6 months.Population: Changes in cognition scores from baseline to month 6.
Montreal Cognitive Assessment (total score range 0-30, higher score = less impairment)
Outcome measures
| Measure |
Active
n=9 Participants
Fenofibrate: 145mg of fenofibrate
|
Placebo
n=1 Participants
Placebo: Placebo
|
|---|---|---|
|
Change in Montreal Cognitive Assesment Score.
Baseline
|
24.222 score on a scale
Standard Deviation 3.701
|
18 score on a scale
|
|
Change in Montreal Cognitive Assesment Score.
Month 3
|
25.778 score on a scale
Standard Deviation 3.866
|
16 score on a scale
|
|
Change in Montreal Cognitive Assesment Score.
Month 6
|
25.667 score on a scale
Standard Deviation 4.093
|
22 score on a scale
|
SECONDARY outcome
Timeframe: Baseline, Month 3 and 6 monthsPopulation: Changes in independence assessment scores from baseline, month 3 to month 6.
Unified Huntington Disease Rating Scale (UHDRS) Behavioral Scale (total score range 0-100 where 100 = 100% independent)
Outcome measures
| Measure |
Active
n=9 Participants
Fenofibrate: 145mg of fenofibrate
|
Placebo
n=1 Participants
Placebo: Placebo
|
|---|---|---|
|
Changes in Unified Huntington Disease Rating Scale - Behavioral
Baseline
|
9.778 score on a scale
Standard Deviation 2.438
|
8 score on a scale
|
|
Changes in Unified Huntington Disease Rating Scale - Behavioral
Month 3
|
8.333 score on a scale
Standard Deviation 3.674
|
9 score on a scale
|
|
Changes in Unified Huntington Disease Rating Scale - Behavioral
Month 6
|
8.444 score on a scale
Standard Deviation 3.575
|
8 score on a scale
|
SECONDARY outcome
Timeframe: Baseline compared to 3 and 6 months.Population: Change in functional assessment scores from baseline, month 3 to month 6.
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)
Outcome measures
| Measure |
Active
n=9 Participants
Fenofibrate: 145mg of fenofibrate
|
Placebo
n=1 Participants
Placebo: Placebo
|
|---|---|---|
|
Change in Functional Assessment Scores
Baseline
|
3.222 score on a scale
Standard Deviation 1.09
|
3 score on a scale
|
|
Change in Functional Assessment Scores
Month 3
|
3.571 score on a scale
Standard Deviation 0.787
|
3 score on a scale
|
|
Change in Functional Assessment Scores
Month 6
|
3.571 score on a scale
Standard Deviation 0.787
|
3 score on a scale
|
Adverse Events
Active
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Placebo
Serious events: 0 serious events
Other events: 0 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
Adverse event data not reported
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place