Clinical trial NCT02736149
Conditions: Pulmonary Arterial Hypertension
Sponsor: Eiger BioPharmaceuticals
Start date: 2016-12-01; Completion date: 2018-08-01
Enrollment: 51 participants; Allocation: NA; Intervention model: SINGLE_GROUP

Brief summary:
Ubenimex is being developed for the treatment of pulmonary arterial hypertension (PAH) (World Health Organization \[WHO\] Group 1) to improve exercise capacity and delay clinical worsening.

This study is a Phase 2, open-label, extension study to evaluate long-term safety and efficacy of ubenimex in patients with PAH (WHO Group 1) who complete Study EIG-UBX-001.

Detailed description:
This study is a Phase 2, open-label, multicenter, extension study to evaluate long-term safety and efficacy of ubenimex in patients with PAH (WHO Group 1) who complete Study EIG-UBX-001 (Study EIG-UBX-001 is a Phase 2, randomized, double-blind, placebo-controlled clinical trial) and meet the eligibility criteria for Study EIG-UBX-002. The primary objective for this study is:

\- to obtain long-term safety and tolerability data for ubenimex (150 mg administered three times daily \[TID\]) in patients with PAH (WHO Group 1).

Arms: arm 1: ubenimex capsules 150 mg three times a day (TID), administered orally, minimum of 24 weeks for all patients.

The maximum anticipated time an individual patient will participate will vary because treatment will continue until the last patient enrolled has received at least 24 weeks of open-label treatment.
Interventions: intervention 1: ubenimex
Intervention descriptions: intervention 1: None

Protocol documents:
Prot_SAP_000.pdf: 
Page 1 of 4 
 
 
 
 
 
 
 
Title: A PHASE 2, OPEN-LABEL, EXTENSION STUDY TO EVALUATE THE 
LONG-TERM SAFETY AND EFFICACY OF UBENIMEX IN PATIENTS 
WITH PULMONARY ARTERIAL HYPERTENSION (WHO GROUP 1) 
 
 
 
NCT02736149 
 
Document date: 03/31/2021 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 
 


Page 2 of 4 
 
Protocol:  
Objectives  
The primary objective for the study is to obtain long-term safety and tolerability data for 
ubenimex in patients with pulmonary arterial hypertension (PAH). 
Methodology 
This was a Phase 2, open-label, multicenter, extension study to evaluate long-term 
safety and efficacy of ubenimex (150 mg three times a day) in patients with PAH who 
completed Study EIG-UBX-001 (a Phase 2, randomized, double-blind, placebo-
controlled clinical trial) and met the eligibility criteria for Study EIG-UBX-002.  
Patients may have qualified to enter this extension study if they completed study drug 
treatment and study procedures through Week 24 in Study EIG-UBX-001 and met the 
entry criteria for Study EIG UBX 002. Data gathered at the Week 24 (End-of-Treatment) 
Visit formed the baseline data for EIG-UBX-002. Treatment in EIG-UBX-002 continued 
until the last patient enrolled received at least 24 weeks of open-label treatment with 
ubenimex and the Sponsor decided to end the study. Clinical assessments were 
performed on Week 0E, where “E” denotes the extension study EIG-UBX-002 (Week 24 
of Study EIG-UBX-001) and on Weeks 4E, 8E, 12E, and 24E, and every 12 weeks 
thereafter until the end of study. The total duration of treatment for each patient varied 
depending on when the patient enrolled and the recruitment rate in Study EIG-UBX-001. 
At the end of the Treatment Period in EIG-UBX-002, patients returned 4 weeks after 
their last dose of ubenimex for a final Follow-up Visit.  
Safety (adverse events [AEs], clinical laboratory tests) was continually monitored 
throughout the study. An independent data safety monitoring board, consisting of two 
expert PAH clinicians and a biostatistician, conducted regular safety reviews. Efficacy 
measures included pulmonary vascular resistance (PVR) and 6 minute walk distance. 
The steady-state pharmacokinetics of ubenimex and metabolite was to be 
characterized.  
Inclusion Criteria 
A patient was included in this study if he or she met all of the following criteria: 
• 
Patients must have completed Study EIG-UBX-001 through Week 24. 
• 
In the opinion of the Principal Investigator, has been generally compliant with 
study requirements during Study EIG-UBX-001. 
• 
Agrees to use a medically acceptable method of contraception throughout the 
entire study period. 
• 
Willing and able to comply with scheduled visits, treatment plans, and laboratory 
tests and other study procedures. 


Page 3 of 4 
 
Exclusion Criteria 
Patients meeting any of the following criteria were excluded from the study: 
• 
Is pregnant or lactating. 
• 
Concurrent regular use of another leukotriene pathway inhibitor. 
• 
Any reason that, in the opinion of the investigator, precludes the patient from 
participating in the study. 
a. Any condition that is unstable or that could jeopardize the safety of the 
patient and his/her compliance in the study 
b. A serious uncontrolled medical disorder/condition that in the opinion of the 
investigator would impair the ability of the patient to receive protocol 
therapy 
• 
An ongoing, drug-related, serious adverse event (SAE). 
• 
Significant/chronic renal insufficiency. 
• 
Transaminases (alanine transaminase, aspartate transaminase) levels >3 × 
upper limit of normal (ULN) and/or bilirubin level >2 × ULN. 
• 
Absolute neutrophil count <1500 mm3. 
• 
Hemoglobin concentration <9 g/dL at Screening. 
Dose and Mode of Administration  
150 mg administered orally three times a day (at 8 ± 2 h intervals), with or without food. 
Duration of Treatment 
Treatment Period: minimum of 24 weeks for all patients. Follow-up Period: 4 weeks. 
The maximum time an individual patient participated varied because treatment 
continued until the last patient enrolled had received at least 24 weeks of open-label 
treatment. 
Criteria for Evaluation 
All endpoints were changes from start of treatment in EIG-UBX-002 to the end of 
treatment in EIG-UBX-002.  
Due to the early termination of the study, only safety information was assessed, and 
treatment-emergent adverse events and SAEs and deaths were evaluated of the 
relatedness of the drug treatment. 
 
 


Page 4 of 4 
 
Statistical Methods 
Following the failure to demonstrate efficacy in EIG-UBX-001, the sponsor terminated 
Study EIG-UBX-002. Only safety data were assessed. 
Safety Set (SS) are patients who received at least one dose of study drug in study. 
Data collected at Week 24 of Study EIG-UBX-001 were used as baseline values for 
Study EIG-UBX-002. An event that had onset in Study EIG-UBX-001 was not 
considered an event in Study EIG-UBX-002 unless it worsened in Study EIG-UBX-002, 
in which case it became a separate event. Adverse events were coded using MedDRA 
version 18.1.