Trial Outcomes & Findings for Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients (NCT NCT02430077)
NCT ID: NCT02430077
Last Updated: 2024-08-27
Results Overview
The primary end-point variable was the change in the liver TG content assessed using proton-density fat fraction mapping by Magnetic Resonance Imaging (MRI).
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
10 participants
Primary outcome timeframe
Baseline, 4 months
Results posted on
2024-08-27
Participant Flow
A total of thirteen subjects were consented in the study. Ten adult female subjects completed the study. Two subjects did not qualify based on the enrollment criteria (screen failed). One subject withdrew due to personal reasons prior to being randomized. Hence only 10 subjects considered enrolled.
Participant milestones
| Measure |
Active Capsule of Obeticholic Acid, Then Placebo
Patient received obeticholic acid (OCA) in the dose of 25 mg/day for a period of 4 months.
Then a washout period of 4 months.
Afterwards, patients received identical placebo in the dose of 25 mg/day for a period of 4 months .
|
Placebo for Obeticholic Acid, Then Active Drug
Patient received placebo in the dose of 25 mg/day for a period of 4 months.
Washout period of 4 months
Thereafter, patients received Obeticholic Acid in the dose of 25 mg/day for a period of 4 months.
|
|---|---|---|
|
First Intervention 4 Months
STARTED
|
5
|
5
|
|
First Intervention 4 Months
COMPLETED
|
5
|
5
|
|
First Intervention 4 Months
NOT COMPLETED
|
0
|
0
|
|
Washout 4 Months
STARTED
|
5
|
5
|
|
Washout 4 Months
COMPLETED
|
5
|
5
|
|
Washout 4 Months
NOT COMPLETED
|
0
|
0
|
|
Second Intervention 4 Months
STARTED
|
5
|
5
|
|
Second Intervention 4 Months
COMPLETED
|
5
|
5
|
|
Second Intervention 4 Months
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Phase 2 Study of Obeticholic Acid for Lipodystrophy Patients
Baseline characteristics by cohort
| Measure |
All Study Participants
n=10 Participants
Patients were randomized to receive Obeticholic acid or identical placebo in the dose of 25 mg/day for a period of 4 months. This was followed by a wash out period of 4 months. Thereafter, they crossed-over to receive Obeticholic acid or identical placebo in the dose of 25 mg/day for a period of 4 months.
Placebo: Identical to Obeticholic Acid - placebo drug
|
|---|---|
|
Age, Continuous
|
37.5 years
STANDARD_DEVIATION 15 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
White
|
10 Participants
n=5 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=5 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=5 Participants
|
|
Liver Fat
|
13 percentage of liver fat
STANDARD_DEVIATION 8 • n=5 Participants
|
PRIMARY outcome
Timeframe: Baseline, 4 monthsThe primary end-point variable was the change in the liver TG content assessed using proton-density fat fraction mapping by Magnetic Resonance Imaging (MRI).
Outcome measures
| Measure |
Baseline (Month 0)
n=10 Participants
Before starting the active drug, Obeticholic Acid.
|
Obeticholic Acid (Month 4)
n=10 Participants
Patient recieved Obeticholic Acid in the dose of 25 mg/day for a period of 4 months.
|
Placebo (Month 0)
n=10 Participants
Before starting the Placebo
|
Placebo (Month 4)
n=10 Participants
Patient received placebo in the dose of 25 mg/day for a period of 4 months.
|
|---|---|---|---|---|
|
Change in the Liver Triglycerides (TG).
|
13.2 percentage of liver triglycerides
Interval 2.8 to 28.5
|
6.4 percentage of liver triglycerides
Interval 2.4 to 18.0
|
12.3 percentage of liver triglycerides
Interval 3.6 to 33.3
|
10.6 percentage of liver triglycerides
Interval 3.4 to 29.3
|
SECONDARY outcome
Timeframe: Baseline, Month 4Change in the serum levels of Triglycerides from baseline to month 4 is being assessed.
Outcome measures
| Measure |
Baseline (Month 0)
n=10 Participants
Before starting the active drug, Obeticholic Acid.
|
Obeticholic Acid (Month 4)
n=10 Participants
Patient recieved Obeticholic Acid in the dose of 25 mg/day for a period of 4 months.
|
Placebo (Month 0)
n=10 Participants
Before starting the Placebo
|
Placebo (Month 4)
n=10 Participants
Patient received placebo in the dose of 25 mg/day for a period of 4 months.
|
|---|---|---|---|---|
|
Change in Serum Triglyceride Levels
|
199.2 mg/dL
Interval 78.0 to 392.0
|
182.3 mg/dL
Interval 102.0 to 309.3
|
169.3 mg/dL
Interval 103.5 to 562.0
|
161.3 mg/dL
Interval 93.0 to 655.0
|
SECONDARY outcome
Timeframe: Baseline, Month 4Change in serum levels of Alanine Aminotransferase from baseline to month 4 is assessed
Outcome measures
| Measure |
Baseline (Month 0)
n=10 Participants
Before starting the active drug, Obeticholic Acid.
|
Obeticholic Acid (Month 4)
n=10 Participants
Patient recieved Obeticholic Acid in the dose of 25 mg/day for a period of 4 months.
|
Placebo (Month 0)
n=10 Participants
Before starting the Placebo
|
Placebo (Month 4)
n=10 Participants
Patient received placebo in the dose of 25 mg/day for a period of 4 months.
|
|---|---|---|---|---|
|
Change in Serum Levels of Alanine Aminotransferase
|
16.3 U/L
Interval 13.5 to 31.0
|
15.8 U/L
Interval 13.0 to 42.5
|
19.0 U/L
Interval 11.0 to 33.5
|
18.5 U/L
Interval 12.6 to 33.0
|
SECONDARY outcome
Timeframe: Baseline, Month4Change in serum levels of Aspartate Aminotransferases from baseline to month 4 is assessed
Outcome measures
| Measure |
Baseline (Month 0)
n=10 Participants
Before starting the active drug, Obeticholic Acid.
|
Obeticholic Acid (Month 4)
n=10 Participants
Patient recieved Obeticholic Acid in the dose of 25 mg/day for a period of 4 months.
|
Placebo (Month 0)
n=10 Participants
Before starting the Placebo
|
Placebo (Month 4)
n=10 Participants
Patient received placebo in the dose of 25 mg/day for a period of 4 months.
|
|---|---|---|---|---|
|
Change in Serum Levels of Aspartate Aminotransferases
|
15.5 U/L
Interval 13.5 to 35.5
|
16.8 U/L
Interval 13.7 to 27.0
|
18.7 U/L
Interval 13.5 to 28.5
|
16.5 U/L
Interval 12.5 to 31.0
|
SECONDARY outcome
Timeframe: Baseline, Month 4Change in serum levels of Gamma-Glutamyl Transpeptidase from baseline to month 4 is assessed
Outcome measures
| Measure |
Baseline (Month 0)
n=10 Participants
Before starting the active drug, Obeticholic Acid.
|
Obeticholic Acid (Month 4)
n=10 Participants
Patient recieved Obeticholic Acid in the dose of 25 mg/day for a period of 4 months.
|
Placebo (Month 0)
n=10 Participants
Before starting the Placebo
|
Placebo (Month 4)
n=10 Participants
Patient received placebo in the dose of 25 mg/day for a period of 4 months.
|
|---|---|---|---|---|
|
Change in Serum Levels of Gamma-Glutamyl Transpeptidase
|
27.5 U/L
Interval 16.3 to 64.0
|
17 U/L
Interval 10.6 to 45.7
|
21.6 U/L
Interval 14.6 to 109.0
|
21.2 U/L
Interval 13.5 to 66.0
|
SECONDARY outcome
Timeframe: Baseline, Month 4Population: Data was not collected
Changes in serum Insulin levels is assessed
Outcome measures
Outcome data not reported
SECONDARY outcome
Timeframe: Baseline, Month 4Population: Data not collected.
Change in hepatic insulin sensitivity is assessed by suppression of hepatic glucose output during the low-dose and high-dose insulin infusions during the euglycemic clamp study.
Outcome measures
Outcome data not reported
Adverse Events
Active Capsule of Obeticholic Acid
Serious events: 0 serious events
Other events: 4 other events
Deaths: 0 deaths
Placebo for Obeticholic Acid
Serious events: 0 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
Adverse event data not reported
Other adverse events
| Measure |
Active Capsule of Obeticholic Acid
n=10 participants at risk
Patient received obeticholic acid (OCA) in the dose of 25 mg/day for a period of 4 months.
Obeticholic Acid: Capsules of obeticholic acid (OCA) in the dose of 25 mg/day for a period of 4 months .
|
Placebo for Obeticholic Acid
n=10 participants at risk
Patient received placebo in the dose of 25 mg/day for a period of 4 months.
Placebo: Identical to Obeticholic Acid - no active ingredient
|
|---|---|---|
|
Skin and subcutaneous tissue disorders
Itching
|
40.0%
4/10 • 4 months for each intervention separated by a 4-month washout period, approximately 12 months total
|
20.0%
2/10 • 4 months for each intervention separated by a 4-month washout period, approximately 12 months total
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place