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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.
Full description
Ubenimex is being developed for the treatment of PAH (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/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.
Enrollment
Sex
Ages
Volunteers
Inclusion criteria
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:
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.
Exclusion criteria
Exclusions Related to Cardiovascular Disease
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.
Exclusions Related to Pulmonary Disease
Newly diagnosed with PAH and not on PAH-specific therapy.
Pulmonary hypertension due to:
Evidence of significant airway and/or parenchymal lung disease.
Chronic infection related to tuberculosis or fungal or mycobacterial disease.
Exclusions Based on Other Medical Conditions
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.
Evidence of active infection requiring intravenous or oral antibiotics within 4 weeks of Screening.
Body mass index ≥35.0 at Screening.
History of obstructive sleep apnea.
History of malignancy within the last 5 years, except nonmelanoma skin cancer and cervical carcinoma in situ treated with curative intent.
Neuropsychiatric disorders/symptoms or psychological conditions.
Pregnancy or breast-feeding
Prior treatment with B cell or lymphocyte-depleting agents (eg, rituximab, Campath)
Exclusions Based on Concomitant Medication Use
Concurrent regular use of another leukotriene pathway inhibitor, including over-the-counter medications or herbal remedies.
Exclusions Based on Laboratory Values
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.
Hepatic dysfunction as defined by Child-Pugh Class B or C
Primary purpose
Allocation
Interventional model
Masking
61 participants in 2 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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