Study to Evaluate Efficacy and Safety of JTT-251 in Participants With Pulmonary Arterial Hypertension (RELIEF-PAH)

Akros Pharma

Status and phase: Withdrawn; Phase 2

Conditions: Pulmonary Arterial Hypertension

Treatments: Drug: Placebo; Drug: JTT-251

Study type: Interventional

Funder types: Industry

Identifiers

NCT03789643

AT251-G-17-006

Details and patient eligibility

About

Study to evaluate the efficacy, safety, tolerability and pharmacokinetics of JTT-251 administered for 24 weeks in participants with pulmonary arterial hypertension (PAH)

Full description

This is a study to evaluate the efficacy, safety, tolerability and pharmacokinetics of JTT-251 administered for 24 weeks in participants with pulmonary arterial hypertension (PAH). Participants completing this study (RELIEF-PAH) will be eligible to enroll in an open-label extension study (RELIEF-PAH OLE) to evaluate the long-term efficacy, safety, tolerability and pharmacokinetics of JTT-251 in participants with PAH.

Sex: All

Ages: 18 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Diagnosis of PAH as classified by idiopathic, heritable, drug and toxin- induced, congenital heart disease or associated with connective tissue disease (i.e., WHO Group 1)
• Clinical diagnosis of PAH confirmed by RHC at any time prior to Visit 1
• WHO functional status of Class II-IV at Visit 1
• Two 6MWD test measurements between 100 and 450 meters with a relative difference of ≤15%. The baseline 6MWD test must be performed at Visit 2 before randomization.
• Have a qualifying RHC performed between Visit 1 and Visit 2
• On stable dose(s) of guideline-directed medical therapy for PAH (endothelin receptor antagonists, phosphodiesterase type-5 (PDE-5) inhibitors, soluble guanylate cyclase stimulators and prostacyclin pathway analogs) for at least 90 days prior to the qualifying RHC

Exclusion criteria

• PAH associated with portal hypertension, human immunodeficiency virus (HIV), schistosomiasis or sickle cell disease as well as participants with pulmonary parenchymal disease or thromboembolic disease
• Known significant left heart disease including: left ventricular dysfunction (i.e., left ventricular ejection fraction <35%); hemodynamically compromising, symptomatic, or severe aortic stenosis, aortic regurgitation, mitral stenosis, mitral regurgitation
• Pulmonary hypertension belonging to WHO groups 2 to 5
• Moderate to severe obstructive lung disease defined as forced expiratory volume in 1 second (FEV1) <55% of predicted value
• Moderate to severe restrictive lung disease defined as total lung capacity (TLC) <60% of predicted value
• Acute decompensated heart failure or hospital admission for worsening PAH symptoms within 30 days prior to the qualifying RHC

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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0 participants in 4 patient groups, including a placebo group

JTT-251 Dose 1

Experimental group

Description:

One dose of study drug by mouth daily for 24 weeks

Treatment:

Drug: JTT-251

JTT-251 Dose 2

Experimental group

Description:

One dose of study drug by mouth daily for 24 weeks

Treatment:

Drug: JTT-251

JTT-251 Dose 3

Experimental group

Description:

One dose of study drug by mouth daily for 24 weeks

Treatment:

Drug: JTT-251

Placebo

Placebo Comparator group

Description:

One dose of study drug by mouth daily for 24 weeks

Treatment:

Drug: Placebo