A Study of Ubenimex in Patients With Pulmonary Arterial Hypertension (WHO Group 1)

NCT02664558 | Completed Phase 2 Results

• 1 other identifier: EIG-UBX-001
• Study Type: interventional
• Target: 61
• Locations: 2 countries
• Sites: 31

Brief Summary

This proof-of-concept study is designed as a Phase 2, multicenter, randomized, double-blind, placebo controlled study comparing ubenimex with placebo in patients with pulmonary arterial hypertension (PAH) (World Health Organization \[WHO\] Group 1) and have a WHO/New York Heart Association (NYHA) Functional Classification (WHO/NYHA-FC) of II or III.

Conditions

Pulmonary Arterial Hypertension

Outcome Measures

Primary Outcomes (1)

• Change in Pulmonary Vascular Resistance (PVR)

  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.

  Baseline to Week 24

Secondary Outcomes (1)

• Change in 6-minute Walk Distance (6MWD)

  Baseline to Week 24

Study Arms (2)

ubenimex

EXPERIMENTAL

ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.

Drug: ubenimex

placebo

PLACEBO COMPARATOR

placebo capsules TID, administered orally for a total of 24 weeks

Other: placebo

Interventions

ubenimex DRUG

Also known as: UBX

ubenimex

placebo OTHER

placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Male or female, 18-75 years old.
• Has a diagnosis of WHO Group 1 PAH.
• Right heart catheterization performed at Screening with results that are:
• Mean pulmonary arterial pressure ≥25 mmHg (at rest) and
• Pulmonary venous hypertension (measured as pulmonary capillary wedge pressure (PCWP) ≤15 mmHg. If PCWP is not available, then mean left atrial pressure or left ventricular end-diastolic pressure ≤15 mmHg in the absence of left atrial obstruction. and
• Pulmonary vascular resistance (PVR) ≥300 dyn•s/cm5 (3.75 Wood units)
• Has WHO/NYHA-FC of II or III.
• Be on stable dose of at least one of the following PAH-specific therapies: endothelin receptor antagonist, an agent acting on the nitric oxide pathway (phosphodiesterase type 5 inhibitor or soluble guanylate cyclase stimulator), and/or a prostacyclin or prostacyclin analog.
• Has a 6-minute walk distance that is ≥150 and ≤500 meters.
• Have a ventilation-perfusion scan that rules out thromboembolic disease.

You may not qualify if:

• History of uncontrolled hypertension
• Persistent hypotension at Screening.
• Evidence or history of left-sided heart disease and/or clinically significant cardiac disease in which pulmonary hypertension is more likely WHO Group 2.
• Acute decompensated heart failure within 1 month of Screening.
• Recent initiation (\<8 weeks from Screening) or planned initiation of cardiopulmonary rehabilitation exercise program.
• Newly diagnosed with PAH and not on PAH-specific therapy.
• Pulmonary hypertension due to:
• Uncorrected congenital systemic-to-pulmonary shunt.
• Pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis
• Persistent pulmonary hypertension of the newborn
• WHO clinical classification Groups 2-5
• Evidence of significant airway and/or parenchymal lung disease.
• Chronic infection related to tuberculosis or fungal or mycobacterial disease.
• Chronic infections including, but not limited to tuberculosis (TB), hepatitis B virus (HBV) or hepatitis C virus (HCV).
• History of portal hypertension or chronic liver disease, including positive serology for infection with HCV and/or HBV.

Sponsors & Collaborators

• Eiger BioPharmaceuticals: lead

Study Sites (31)

University of Alabama at Birmingham

Birmingham, Alabama, 35233, United States

Location

California Heart Center Foundation, An Affiliate of Cedars-Sinai Heart Institute, Cedars-Sinai Medical Care Foundation

Beverly Hills, California, 90211, United States

Location

UCSD Medical Center

La Jolla, California, 92103, United States

Location

Stanford University Medical Center

Stanford, California, 94305, United States

Location

University of Colorado Denver

Aurora, Colorado, 80045, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Cleveland Clinic, Florida

Weston, Florida, 33331, United States

Location

Kentuckiana Pulmonary Associates

Louisville, Kentucky, 40202, United States

Location

Chest Medicine Associates

South Portland, Maine, 04106, United States

Location

Johns Hopkins University, Pulmonary and Critical Care Medicine

Baltimore, Maryland, 21205, United States

Location

Tufts Medical Center

Boston, Massachusetts, 02111, United States

Location

Brigham and Women's Hospital

Boston, Massachusetts, 02115, United States

Location

Boston University School of Medicine

Boston, Massachusetts, 02118, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Mayo Clinic College of Medicine

Rochester, Minnesota, 55905, United States

Location

Washington University

St Louis, Missouri, 63130, United States

Location

Weill Cornell Medicine

New York, New York, 10065, United States

Location

The University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Duke University Medical Center

Durham, North Carolina, 27710, United States

Location

Cleveland Clinic Respiratory Institute

Cleveland, Ohio, 44195, United States

Location

University of Pennsylvania

Philadelphia, Pennsylvania, 19104, United States

Location

Allegheny General Hospital

Pittsburgh, Pennsylvania, 15212, United States

Location

University of Pennsylvania Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Alpert Medical School of Brown University Rhode Island Hospital

Providence, Rhode Island, 02903, United States

Location

Vanderbilt University Medical Center

Nashville, Tennessee, 37232, United States

Location

UT Southwestern Medical Center

Dallas, Texas, 75390, United States

Location

Houston Methodist Hospital

Houston, Texas, 77030, United States

Location

University Texas Health Science Center

San Antonio, Texas, 78229, United States

Location

London Health Sciences Centre

London, Ontario, N6A 5W9, Canada

Location

University of Ottawa Heart Institute

Ottawa, Ontario, K1Y 4W7, Canada

Location

Toronto General Hospital

Toronto, Ontario, M5G 2N2, Canada

Location

Related Publications (2)

• 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.

  PMID: 23986401 BACKGROUND

• 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.

  PMID: 26558820 BACKGROUND

MeSH Terms

Conditions

Pulmonary Arterial Hypertension

Interventions

ubenimex

Condition Hierarchy (Ancestors)

Hypertension, Pulmonary; Lung Diseases; Respiratory Tract Diseases

Results Point of Contact

• Title: Senior VP, Clinical Development
• Organization: Eiger BioPharmaceuticals, Inc.

info@eigerbio.com

1-650-618-1621

Publication Agreements

• PI is Sponsor Employee: No
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 21, 2016
• First Posted: January 27, 2016
• Study Start: April 1, 2016
• Primary Completion: November 1, 2017
• Study Completion: January 1, 2018
• Last Updated: March 6, 2023
• Results First Posted: March 6, 2023

Record last verified: 2023-03

Locations

CA (3); US (28)