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

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ClinicalTrials.gov Identifier: NCT02664558
Recruitment Status : Completed
First Posted : January 27, 2016
Last Update Posted : November 15, 2018
Information provided by (Responsible Party):
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 proof-of-concept study is designed as a Phase 2, multicenter, randomized, double-blind, placebo controlled study comparing ubenimex with placebo in patients with PAH (WHO Group 1) and have a WHO/New York Heart Association (NYHA) Functional Classification (WHO/NYHA-FC) of II or III. The Primary Objectives for the study are:

  • To evaluate the efficacy of ubenimex in patients with PAH (WHO Group 1).
  • To evaluate the safety and tolerability of ubenimex in patients with WHO Group 1 PAH.

Condition or disease Intervention/treatment Phase
Pulmonary Arterial Hypertension Drug: ubenimex Other: placebo Phase 2

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 61 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 2, Randomized, Double-BLInd, Placebo- Controlled Study of UBEnimex in Patients With Pulmonary ARTerial HYpertension (WHO Group 1) (LIBERTY)
Study Start Date : April 2016
Actual Primary Completion Date : November 2017
Actual Study Completion Date : January 2018

Arm Intervention/treatment
Experimental: ubenimex
ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.
Drug: ubenimex
Placebo Comparator: placebo
placebo capsules TID, administered orally for a total of 24 weeks
Other: placebo

Primary Outcome Measures :
  1. Change in pulmonary vascular resistance (PVR). [ Time Frame: Week 24 ]

Secondary Outcome Measures :
  1. Change in exercise capacity as determined by 6-minute walk distance (6MWD). [ Time Frame: Week 24 ]
  2. Change in Borg dyspnea score. [ Time Frame: Week 24 ]
  3. Change in World Health Organization/New York Heart Association Functional Classification [ Time Frame: Week 24 ]
  4. Time to clinical worsening (TTCW) will be assessed and will be evaluated as the number of days between the first dose of study drug and the occurrence of a predefined clinical worsening event [ Time Frame: 24 weeks ]
  5. QoL questionnaire (CAMPHOR, Cambridge Pulmonary Hypertension Outcome Review) [ Time Frame: week 24 ]
  6. Change in disease biomarkers brain natriuretic peptide (BNP) /N-terminal pro-brain natriuretic peptide (NT-proBNP ) [ Time Frame: week 24 ]

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  1. Male or female, 18-75 years old.
  2. Has a diagnosis of WHO Group 1 PAH.
  3. Right heart catheterization performed at Screening with results that are:

    1. Mean pulmonary arterial pressure ≥25 mmHg (at rest) and
    2. 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
    3. Pulmonary vascular resistance (PVR) ≥300 dyn•s/cm5 (3.75 Wood units)
  4. Has WHO/NYHA-FC of II or III.
  5. 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.
  6. Has a 6-minute walk distance that is ≥150 and ≤500 meters.
  7. Have a ventilation-perfusion scan that rules out thromboembolic disease.

Exclusion Criteria:

Exclusions Related to Cardiovascular Disease

  1. History of uncontrolled hypertension
  2. Persistent hypotension at Screening.
  3. Evidence or history of left-sided heart disease and/or clinically significant cardiac disease in which pulmonary hypertension is more likely WHO Group 2.
  4. Acute decompensated heart failure within 1 month of Screening.
  5. Recent initiation (<8 weeks from Screening) or planned initiation of cardiopulmonary rehabilitation exercise program.

    Exclusions Related to Pulmonary Disease

  6. Newly diagnosed with PAH and not on PAH-specific therapy.
  7. Pulmonary hypertension due to:

    1. Uncorrected congenital systemic-to-pulmonary shunt.
    2. Pulmonary veno-occlusive disease and/or pulmonary capillary hemangiomatosis
    3. Persistent pulmonary hypertension of the newborn
    4. WHO clinical classification Groups 2-5
  8. Evidence of significant airway and/or parenchymal lung disease.
  9. Chronic infection related to tuberculosis or fungal or mycobacterial disease.

    Exclusions Based on Other Medical Conditions

  10. Chronic infections including, but not limited to tuberculosis (TB), hepatitis B virus (HBV) or hepatitis C virus (HCV).
  11. History of portal hypertension or chronic liver disease, including positive serology for infection with HCV and/or HBV.
  12. Evidence of active infection requiring intravenous or oral antibiotics within 4 weeks of Screening.
  13. Body mass index ≥35.0 at Screening.
  14. History of obstructive sleep apnea.
  15. History of malignancy within the last 5 years, except nonmelanoma skin cancer and cervical carcinoma in situ treated with curative intent.
  16. Neuropsychiatric disorders/symptoms or psychological conditions.
  17. Pregnancy or breast-feeding
  18. Prior treatment with B cell or lymphocyte-depleting agents (eg, rituximab, Campath)

    Exclusions Based on Concomitant Medication Use

  19. Concurrent regular use of another leukotriene pathway inhibitor, including over-the-counter medications or herbal remedies.

    Exclusions Based on Laboratory Values

  20. Significant/chronic renal insufficiency.
  21. Transaminases (alanine transaminase, aspartate transaminase) levels >3 × upper limit of normal (ULN) and/or bilirubin level >2 × ULN.
  22. Absolute neutrophil count <1500 mm3.
  23. Hemoglobin concentration <9 g/dL at Screening.
  24. Hepatic dysfunction as defined by Child-Pugh Class B or C

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02664558

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Sponsors and Collaborators
Eiger BioPharmaceuticals
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Responsible Party: Eiger BioPharmaceuticals
ClinicalTrials.gov Identifier: NCT02664558    
Other Study ID Numbers: EIG-UBX-001
First Posted: January 27, 2016    Key Record Dates
Last Update Posted: November 15, 2018
Last Verified: November 2018
Additional relevant MeSH terms:
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Pulmonary Arterial Hypertension
Familial Primary Pulmonary Hypertension
Vascular Diseases
Cardiovascular Diseases
Hypertension, Pulmonary
Lung Diseases
Respiratory Tract Diseases
Adjuvants, Immunologic
Immunologic Factors
Physiological Effects of Drugs
Anti-Bacterial Agents
Anti-Infective Agents
Antibiotics, Antineoplastic
Antineoplastic Agents
Protease Inhibitors
Enzyme Inhibitors
Molecular Mechanisms of Pharmacological Action
Anti-HIV Agents
Anti-Retroviral Agents
Antiviral Agents