Open-Label Extension Study of Ubenimex in Patients With Pulmonary Arterial Hypertension (WHO Group 1)

Phase 2

Terminated

Conditions: Pulmonary Arterial Hypertension

Interventions: Drug: ubenimex

Registration Number: NCT02736149

Lead Sponsor: Eiger BioPharmaceuticals

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).

Recruitment & Eligibility

Status: TERMINATED

Sex: All

Target Recruitment: 51

Inclusion 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.

Exclusion Criteria

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.
1. Any condition that is unstable or that could jeopardize the safety of the patient and his/her compliance in the study
2. 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.

Study & Design

Study Type: INTERVENTIONAL

Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
ubenimex	ubenimex	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.

Primary Outcome Measures

Name	Time	Method
Treatment-emergent Adverse Events (TEAEs)	At least 24 weeks of open-label treatment with ubenimex followed by 4 weeks follow-up	Number of Patients with TEAEs collected for all patients and recorded on the Adverse Event Case Report Form

Secondary Outcome Measures

Name	Time	Method

Trial Locations

Locations (28): Harbor - UCLA Medical Center
🇺🇸
Torrance, California, United States
Boston University School of Medicine
🇺🇸
Boston, Massachusetts, United States
University of Pennsylvania
🇺🇸
Philadelphia, Pennsylvania, United States
University of Pennsylvania Medical Center
🇺🇸
Pittsburgh, Pennsylvania, United States
Allegheny General Hospital
🇺🇸
Pittsburgh, Pennsylvania, United States
University of Ottawa Heart Institute
🇨🇦
Ottawa, Ontario, Canada
Houston Methodist Hospital
🇺🇸
Houston, Texas, United States
Vanderbilt University Medical Center
🇺🇸
Nashville, Tennessee, United States
University of Colorado Denver
🇺🇸
Aurora, Colorado, United States
California Heart Center Foundation, An Affiliate of Cedars-Sinai Heart Institute, Cedars-Sinai Medical Care Foundation
🇺🇸
Beverly Hills, California, United States
Cleveland Clinic, Florida
🇺🇸
Weston, Florida, United States
UCSD Medical Center
🇺🇸
La Jolla, California, United States
Stanford University Medical Center
🇺🇸
Palo Alto, California, United States
Johns Hopkins University, Pulmonary and Critical Care Medicine
🇺🇸
Baltimore, Maryland, United States
Kentuckiana Pulmonary Associates
🇺🇸
Louisville, Kentucky, United States
Chest Medicine Associates
🇺🇸
South Portland, Maine, United States
Tufts Medical Center
🇺🇸
Boston, Massachusetts, United States
Brigham and Women's Hospital
🇺🇸
Boston, Massachusetts, United States
Washington University
🇺🇸
Saint Louis, Missouri, United States
Mayo Clinic College of Medicine
🇺🇸
Rochester, Minnesota, United States
Weill Cornell Medicine
🇺🇸
New York, New York, United States
Cleveland Clinic Respiratory Institute
🇺🇸
Cleveland, Ohio, United States
Alpert Medical School of Brown University Rhode Island Hospital
🇺🇸
Providence, Rhode Island, United States
UT Southwestern Medical Center
🇺🇸
Dallas, Texas, United States
University Texas Health Science Center
🇺🇸
San Antonio, Texas, United States
London Health Sciences Centre
🇨🇦
London, Ontario, Canada
Duke University Medical Center
🇺🇸
Durham, North Carolina, United States
University of Michigan
🇺🇸
Ann Arbor, Michigan, United States