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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
Sponsor:
Information provided by (Responsible Party):
Eiger BioPharmaceuticals

Tracking Information
First Submitted Date  ICMJE January 21, 2016
First Posted Date  ICMJE January 27, 2016
Last Update Posted Date November 15, 2018
Study Start Date  ICMJE April 2016
Actual Primary Completion Date November 2017   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: January 26, 2016)
Change in pulmonary vascular resistance (PVR). [ Time Frame: Week 24 ]
Original Primary Outcome Measures  ICMJE Same as current
Change History Complete list of historical versions of study NCT02664558 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures  ICMJE
 (submitted: January 26, 2016)
  • Change in exercise capacity as determined by 6-minute walk distance (6MWD). [ Time Frame: Week 24 ]
  • Change in Borg dyspnea score. [ Time Frame: Week 24 ]
  • Change in World Health Organization/New York Heart Association Functional Classification [ Time Frame: Week 24 ]
  • 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 ]
  • QoL questionnaire (CAMPHOR, Cambridge Pulmonary Hypertension Outcome Review) [ Time Frame: week 24 ]
  • Change in disease biomarkers brain natriuretic peptide (BNP) /N-terminal pro-brain natriuretic peptide (NT-proBNP ) [ Time Frame: week 24 ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Study of Ubenimex in Patients With Pulmonary Arterial Hypertension (WHO Group 1)
Official Title  ICMJE A Phase 2, Randomized, Double-BLInd, Placebo- Controlled Study of UBEnimex in Patients With Pulmonary ARTerial HYpertension (WHO Group 1) (LIBERTY)
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.
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Condition  ICMJE Pulmonary Arterial Hypertension
Intervention  ICMJE
  • Drug: ubenimex
  • Other: placebo
Study Arms  ICMJE
  • Experimental: ubenimex
    ubenimex capsules 150 mg three times a day (TID), administered orally for a total of 24 weeks.
    Intervention: Drug: ubenimex
  • Placebo Comparator: placebo
    placebo capsules TID, administered orally for a total of 24 weeks
    Intervention: Other: placebo
Publications *

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Completed
Actual Enrollment  ICMJE
 (submitted: July 5, 2017)
61
Original Estimated Enrollment  ICMJE
 (submitted: January 26, 2016)
45
Actual Study Completion Date  ICMJE January 2018
Actual Primary Completion Date November 2017   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 18 Years to 75 Years   (Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada,   United States
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT02664558
Other Study ID Numbers  ICMJE EIG-UBX-001
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product Not Provided
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Eiger BioPharmaceuticals
Study Sponsor  ICMJE Eiger BioPharmaceuticals
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Eiger BioPharmaceuticals
Verification Date November 2018

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP