Open-Label Extension Study of Ubenimex in Patients With Pulmonary Arterial Hypertension (WHO Group 1)

Study Overview
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Brief Summary
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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
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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).

Official Title
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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)

Conditions
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Pulmonary Arterial Hypertension

Intervention / Treatment
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* Drug: ubenimex


Participation Criteria
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Eligibility Criteria
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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. Any condition that is unstable or that could jeopardize the safety of the patient and his/her compliance in the study 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.

Ages Eligible for Study
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Minimum Age: 18 Years
Maximum Age: 75 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: ubenimex<br>ubenimex capsules 150 mg three times a day (TID), administered orally, minimum of 24 weeks for all patients. The maximum anticipated time an individual patient will participate will vary because treatment will continue until the last patient enrolled has received at least 24 weeks of open-label treatment. | Drug: ubenimex<br> <br> * Other names: UBX;|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Treatment-emergent Adverse Events (TEAEs) | Number of Patients with TEAEs collected for all patients and recorded on the Adverse Event Case Report Form | At least 24 weeks of open-label treatment with ubenimex followed by 4 weeks follow-up |