Trial Outcomes & Findings for A Study of Ubenimex in Patients With Pulmonary Arterial Hypertension (WHO Group 1) (NCT NCT02664558)
NCT ID: NCT02664558
Last Updated: 2023-03-06
Results Overview
Change from Baseline at End of Treatment in PVR Using Worst Case Imputation in the Modified Intent to Treat Population. PVR was assessed by hemodynamic measurements obtained via right heart catheterization.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
61 participants
Primary outcome timeframe
Baseline to Week 24
Results posted on
2023-03-06
Participant Flow
Participant milestones
| Measure |
Ubenimex
ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.
ubenimex
|
Placebo
placebo capsules TID, administered orally for a total of 24 weeks
placebo
|
|---|---|---|
|
Overall Study
STARTED
|
41
|
20
|
|
Overall Study
COMPLETED
|
36
|
18
|
|
Overall Study
NOT COMPLETED
|
5
|
2
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
A Study of Ubenimex in Patients With Pulmonary Arterial Hypertension (WHO Group 1)
Baseline characteristics by cohort
| Measure |
Ubenimex
n=41 Participants
ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.
ubenimex
|
Placebo
n=20 Participants
placebo capsules TID, administered orally for a total of 24 weeks
placebo
|
Total
n=61 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
49.9 years
n=99 Participants
|
53.0 years
n=107 Participants
|
50.9 years
n=206 Participants
|
|
Sex: Female, Male
Female
|
36 Participants
n=99 Participants
|
15 Participants
n=107 Participants
|
51 Participants
n=206 Participants
|
|
Sex: Female, Male
Male
|
5 Participants
n=99 Participants
|
5 Participants
n=107 Participants
|
10 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
|
36 Participants
n=99 Participants
|
18 Participants
n=107 Participants
|
54 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
|
5 Participants
n=99 Participants
|
2 Participants
n=107 Participants
|
7 Participants
n=206 Participants
|
|
Mean pulmonary vascular resistance (PVR)
|
649.9 dyn•s/cm^5
STANDARD_DEVIATION 315.8 • n=99 Participants
|
624.4 dyn•s/cm^5
STANDARD_DEVIATION 217.1 • n=107 Participants
|
641.57 dyn•s/cm^5
STANDARD_DEVIATION 217.1 • n=206 Participants
|
PRIMARY outcome
Timeframe: Baseline to Week 24Population: Modified Intent to Treat Population: i.e., all randomized patients who received at least one dose of study drug and had a baseline assessment for the respective endpoint.
Change from Baseline at End of Treatment in PVR Using Worst Case Imputation in the Modified Intent to Treat Population. PVR was assessed by hemodynamic measurements obtained via right heart catheterization.
Outcome measures
| Measure |
Ubenimex
n=41 Participants
ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.
ubenimex
|
Placebo
n=20 Participants
placebo capsules TID, administered orally for a total of 24 weeks
placebo
|
|---|---|---|
|
Change in Pulmonary Vascular Resistance (PVR)
|
73.1 dyn.sec/cm5
Standard Deviation 271.6
|
46.6 dyn.sec/cm5
Standard Deviation 256.8
|
SECONDARY outcome
Timeframe: Baseline to Week 24Population: Modified intention-to-treat population: i.e., all randomized patients who received at least one dose of study drug and had a baseline assessment for the respective endpoint.
Change in exercise capacity from baseline to Week 24 as determined by the 6MWD
Outcome measures
| Measure |
Ubenimex
n=41 Participants
ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.
ubenimex
|
Placebo
n=20 Participants
placebo capsules TID, administered orally for a total of 24 weeks
placebo
|
|---|---|---|
|
Change in 6-minute Walk Distance (6MWD)
|
-20.0 meters
Standard Error 12.79
|
4.2 meters
Standard Error 17.38
|
Adverse Events
Ubenimex
Serious events: 9 serious events
Other events: 39 other events
Deaths: 0 deaths
Placebo
Serious events: 2 serious events
Other events: 18 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Ubenimex
n=41 participants at risk
ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.
ubenimex
|
Placebo
n=20 participants at risk
placebo capsules TID, administered orally for a total of 24 weeks
placebo
|
|---|---|---|
|
General disorders
Complication associated with device
|
2.4%
1/41 • 7 months
|
5.0%
1/20 • 7 months
|
|
Infections and infestations
Pneumonia
|
0.00%
0/41 • 7 months
|
5.0%
1/20 • 7 months
|
|
General disorders
Diarrhoea infectious
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Gastrointestinal disorders
Pancreatitis
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Infections and infestations
Tracheobronchitis
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Hypoxia
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Infections and infestations
Upper respiratory tract infection
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Skin and subcutaneous tissue disorders
Cellulitis
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Infections and infestations
Clostridium difficile infection
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Renal and urinary disorders
Renal colic
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Reproductive system and breast disorders
Progressive pulmonary hypertension
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Cardiac disorders
Cardiac failure
|
2.4%
1/41 • 7 months
|
0.00%
0/20 • 7 months
|
Other adverse events
| Measure |
Ubenimex
n=41 participants at risk
ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.
ubenimex
|
Placebo
n=20 participants at risk
placebo capsules TID, administered orally for a total of 24 weeks
placebo
|
|---|---|---|
|
Gastrointestinal disorders
Diarrhea
|
26.8%
11/41 • 7 months
|
25.0%
5/20 • 7 months
|
|
Gastrointestinal disorders
Nausea
|
22.0%
9/41 • 7 months
|
25.0%
5/20 • 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
19.5%
8/41 • 7 months
|
0.00%
0/20 • 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Upper respiratory tract infection
|
17.1%
7/41 • 7 months
|
30.0%
6/20 • 7 months
|
|
Nervous system disorders
Headache
|
12.2%
5/41 • 7 months
|
30.0%
6/20 • 7 months
|
|
Metabolism and nutrition disorders
Hypokalemia
|
12.2%
5/41 • 7 months
|
5.0%
1/20 • 7 months
|
|
Respiratory, thoracic and mediastinal disorders
Sinusitis
|
12.2%
5/41 • 7 months
|
0.00%
0/20 • 7 months
|
Additional Information
Senior VP, Clinical Development
Eiger BioPharmaceuticals, Inc.
Phone: 1-650-618-1621
Email: [email protected]
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place