Clinical trial NCT02736149: Open-Label Extension Study of Ubenimex in Patients With Pulmonary Arterial Hypertension (WHO Group 1)
Official 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)
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Pulmonary Arterial Hypertension
Lead sponsor: Eiger BioPharmaceuticals
Sponsor class: INDUSTRY
First posted: 2016-04-13
Enrollment: 51
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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).

Eligibility criteria: 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.

Primary outcomes:
measure: Treatment-emergent Adverse Events (TEAEs)
timeFrame: At least 24 weeks of open-label treatment with ubenimex followed by 4 weeks follow-up
Secondary outcomes:
Other outcomes:
Locations:
city: Beverly Hills
country: United States
facility: California Heart Center Foundation, An Affiliate of Cedars-Sinai Heart Institute, Cedars-Sinai Medical Care Foundation
state: California
city: La Jolla
country: United States
facility: UCSD Medical Center
state: California
city: Palo Alto
country: United States
facility: Stanford University Medical Center
state: California
city: Torrance
country: United States
facility: Harbor - UCLA Medical Center
state: California
city: Aurora
country: United States
facility: University of Colorado Denver
state: Colorado
city: Weston
country: United States
facility: Cleveland Clinic, Florida
state: Florida
city: Louisville
country: United States
facility: Kentuckiana Pulmonary Associates
state: Kentucky
city: South Portland
country: United States
facility: Chest Medicine Associates
state: Maine
city: Baltimore
country: United States
facility: Johns Hopkins University, Pulmonary and Critical Care Medicine
state: Maryland
city: Boston
country: United States
facility: Tufts Medical Center
state: Massachusetts
city: Boston
country: United States
facility: Brigham and Women's Hospital
state: Massachusetts
city: Boston
country: United States
facility: Boston University School of Medicine
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Rochester
country: United States
facility: Mayo Clinic College of Medicine
state: Minnesota
city: Saint Louis
country: United States
facility: Washington University
state: Missouri
city: New York
country: United States
facility: Weill Cornell Medicine
state: New York
city: Durham
country: United States
facility: Duke University Medical Center
state: North Carolina
city: Cleveland
country: United States
facility: Cleveland Clinic Respiratory Institute
state: Ohio
city: Philadelphia
country: United States
facility: University of Pennsylvania
state: Pennsylvania
city: Pittsburgh
country: United States
facility: Allegheny General Hospital
state: Pennsylvania
city: Pittsburgh
country: United States
facility: University of Pennsylvania Medical Center
state: Pennsylvania
city: Providence
country: United States
facility: Alpert Medical School of Brown University Rhode Island Hospital
state: Rhode Island
city: Nashville
country: United States
facility: Vanderbilt University Medical Center
state: Tennessee
city: Dallas
country: United States
facility: UT Southwestern Medical Center
state: Texas
city: Houston
country: United States
facility: Houston Methodist Hospital
state: Texas
city: San Antonio
country: United States
facility: University Texas Health Science Center
state: Texas
city: London
country: Canada
facility: London Health Sciences Centre
state: Ontario
city: Ottawa
country: Canada
facility: University of Ottawa Heart Institute
state: Ontario
Study officials:
References:
citation: Tian W, Jiang X, Tamosiuniene R, Sung YK, Qian J, Dhillon G, Gera L, Farkas L, Rabinovitch M, Zamanian RT, Inayathullah M, Fridlib M, Rajadas J, Peters-Golden M, Voelkel NF, Nicolls MR. Blocking macrophage leukotriene b4 prevents endothelial injury and reverses pulmonary hypertension. Sci Transl Med. 2013 Aug 28;5(200):200ra117. doi: 10.1126/scitranslmed.3006674.
citation: Qian J, Tian W, Jiang X, Tamosiuniene R, Sung YK, Shuffle EM, Tu AB, Valenzuela A, Jiang S, Zamanian RT, Fiorentino DF, Voelkel NF, Peters-Golden M, Stenmark KR, Chung L, Rabinovitch M, Nicolls MR. Leukotriene B4 Activates Pulmonary Artery Adventitial Fibroblasts in Pulmonary Hypertension. Hypertension. 2015 Dec;66(6):1227-1239. doi: 10.1161/HYPERTENSIONAHA.115.06370. Epub 2015 Oct 5.