Trial Outcomes & Findings for Efficacy, Safety and Tolerability of ISIS 304801 in People With Partial Lipodystrophy With an Open-Label Extension (NCT NCT02639286)
NCT ID: NCT02639286
Last Updated: 2020-10-20
Results Overview
Change from baseline to 16 weeks in log 10 fasting triglycerides
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
5 participants
Primary outcome timeframe
Baseline and 16 weeks
Results posted on
2020-10-20
Participant Flow
Participant milestones
| Measure |
Placebo
Placebo administered via SC
|
ISIS 304801
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Overall Study
STARTED
|
3
|
2
|
|
Overall Study
COMPLETED
|
3
|
2
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Efficacy, Safety and Tolerability of ISIS 304801 in People With Partial Lipodystrophy With an Open-Label Extension
Baseline characteristics by cohort
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
Total
n=5 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Sex: Female, Male
Male
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
0 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
1 Participants
n=206 Participants
|
|
Age, Continuous
|
43.7 Years
STANDARD_DEVIATION 9.0 • n=99 Participants
|
32.0 Years
STANDARD_DEVIATION 12.7 • n=107 Participants
|
39 Years
STANDARD_DEVIATION 11 • n=206 Participants
|
|
Sex: Female, Male
Female
|
3 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
3 Participants
n=99 Participants
|
1 Participants
n=107 Participants
|
4 Participants
n=206 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Asian
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Black or African American
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
White
|
3 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
5 Participants
n=206 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=99 Participants
|
0 Participants
n=107 Participants
|
0 Participants
n=206 Participants
|
|
Region of Enrollment
United States
|
3 participants
n=99 Participants
|
2 participants
n=107 Participants
|
5 participants
n=206 Participants
|
|
Log triglyceride (mg/dL)
|
2.6 log mg/dL
STANDARD_DEVIATION 0.3 • n=99 Participants
|
2.7 log mg/dL
STANDARD_DEVIATION 0.0 • n=107 Participants
|
2.6 log mg/dL
STANDARD_DEVIATION 0.2 • n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline and 16 weeksChange from baseline to 16 weeks in log 10 fasting triglycerides
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change in Log 10 Fasting Triglycerides.
|
0.1 log 10 mg/dl
Standard Deviation 0.2
|
-0.2 log 10 mg/dl
Standard Deviation 0.4
|
SECONDARY outcome
Timeframe: Baseline and 16 weeksChange in lipolysis rate measured using stable isotope tracers (Glycerol rate of appearance) (mg/kgLBM/min)
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change in Lipolysis Rate (Glycerol)
|
0.1 mg/kgLBM/min
Standard Deviation 0.6
|
0.3 mg/kgLBM/min
Standard Deviation 2.0
|
SECONDARY outcome
Timeframe: Baseline and 16 weeksChange in lipolysis rate measured using stable isotope tracers (Palmitate rate of appearance) measured in milligrams per kilogram lean body mass per minute (mg/kgLBM/min)
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change in Lipolysis Rate (Palmitate)
|
0.0 mg/kgLBM/min
Standard Deviation 0.1
|
-0.2 mg/kgLBM/min
Standard Deviation 0.0
|
SECONDARY outcome
Timeframe: Baseline and 16 weeksChange from baseline in hepatic steatosis (magnetic resonance spectroscopy) (mL)
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change From Baseline in Liver Volume
|
-114.5 mL
Standard Deviation 384.2
|
129.2 mL
Standard Deviation 145
|
SECONDARY outcome
Timeframe: Baseline and 16 weeksChange from baseline in hepatic steatosis via magnetic resonance spectroscopy
Outcome measures
| Measure |
Placebo
n=2 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change From Baseline in Hepatic Steatosis
|
4.6 percent
Standard Deviation 14.9
|
-3.8 percent
Standard Deviation 0.92
|
SECONDARY outcome
Timeframe: Baseline and 16 weeksLipoprotein lipase activity in plasma is measured using blood samples obtained 10 minutes after intravenous infusion of 60 units/kg of unfractionated heparin.
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change in Lipoprotein Lipase Activity
|
-1.6 nmol FFA/min
Standard Deviation 1.6
|
-3.2 nmol FFA/min
Standard Deviation 2.0
|
SECONDARY outcome
Timeframe: Baseline and 16 weeksChange in total body insulin sensitivity using the hyperinsulinemic euglycemic clamp (mg/kgLBM/min)
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change in Total Body Insulin Sensitivity
|
-0.1 mg/kgLBM/min
Standard Deviation 2.0
|
0.4 mg/kgLBM/min
Standard Deviation 1.5
|
SECONDARY outcome
Timeframe: Baseline and 16 weeksChange in hemoglobin A1c (HbA1c) (%)
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change in HbA1c
|
0.6 Percent
Standard Deviation 0.3
|
0.7 Percent
Standard Deviation 0.2
|
SECONDARY outcome
Timeframe: Baseline and 16 weeksChange in fasting plasma glucose in mg/dL
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Change in Fasting Plasma Glucose
|
-47 mg/dL
Standard Deviation 77
|
-71 mg/dL
Standard Deviation 100
|
SECONDARY outcome
Timeframe: 16 weeksMeasured via a non-compartmental plasma PK analysis of ISIS 304801
Outcome measures
| Measure |
Placebo
n=3 Participants
Placebo administered via SC
|
ISIS 304801
n=2 Participants
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Plasma ISIS 304801 Level
|
0 ng/mL
Standard Error 0
|
34.5 ng/mL
Standard Error 21.5
|
Adverse Events
Placebo
Serious events: 0 serious events
Other events: 3 other events
Deaths: 0 deaths
ISIS 304801
Serious events: 1 serious events
Other events: 2 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=3 participants at risk
Placebo administered via SC
|
ISIS 304801
n=2 participants at risk
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Gastrointestinal disorders
Acute Pancreatitis
|
0.00%
0/3 • 16 weeks of treatment
|
50.0%
1/2 • 16 weeks of treatment
|
Other adverse events
| Measure |
Placebo
n=3 participants at risk
Placebo administered via SC
|
ISIS 304801
n=2 participants at risk
300 mg of ISIS 304801 administered as SC
|
|---|---|---|
|
Investigations
Injection site reactions
|
0.00%
0/3 • 16 weeks of treatment
|
100.0%
2/2 • 16 weeks of treatment
|
|
Hepatobiliary disorders
Elevated liver enzymes
|
33.3%
1/3 • 16 weeks of treatment
|
50.0%
1/2 • 16 weeks of treatment
|
|
Nervous system disorders
Headache
|
0.00%
0/3 • 16 weeks of treatment
|
50.0%
1/2 • 16 weeks of treatment
|
|
Investigations
IV infiltration
|
33.3%
1/3 • 16 weeks of treatment
|
0.00%
0/2 • 16 weeks of treatment
|
|
Renal and urinary disorders
Urinary tract infection
|
0.00%
0/3 • 16 weeks of treatment
|
50.0%
1/2 • 16 weeks of treatment
|
|
Infections and infestations
Influenza
|
0.00%
0/3 • 16 weeks of treatment
|
50.0%
1/2 • 16 weeks of treatment
|
|
Metabolism and nutrition disorders
Hyperglycemia
|
0.00%
0/3 • 16 weeks of treatment
|
50.0%
1/2 • 16 weeks of treatment
|
|
Blood and lymphatic system disorders
Pancytopenia
|
0.00%
0/3 • 16 weeks of treatment
|
50.0%
1/2 • 16 weeks of treatment
|
|
Respiratory, thoracic and mediastinal disorders
URI
|
33.3%
1/3 • 16 weeks of treatment
|
0.00%
0/2 • 16 weeks of treatment
|
|
Gastrointestinal disorders
Nausea
|
33.3%
1/3 • 16 weeks of treatment
|
0.00%
0/2 • 16 weeks of treatment
|
|
Gastrointestinal disorders
Vomiting
|
33.3%
1/3 • 16 weeks of treatment
|
0.00%
0/2 • 16 weeks of treatment
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place