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

Phase 2

Withdrawn

• Conditions: Pulmonary Arterial Hypertension
• Interventions: Drug: JTT-251; Drug: Placebo

• Registration Number: NCT03789643
• Lead Sponsor: Akros Pharma Inc.

Brief Summary

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

Detailed 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.

Study Timeline

• Study First Submitted: 2018-12-26
• Study First Submitted QC: 2018-12-26
• Study First Posted: 2018-12-28
• Study Start: 2019-03
• Status Verified: 2019-05
• Last Update Submitted: 2019-05-01
• Last Update Posted: 2019-05-03
• Primary Completion: 2021-03
• Study Completion: 2021-06

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

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

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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

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Study & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
JTT-251 Dose 2	JTT-251	One dose of study drug by mouth daily for 24 weeks
JTT-251 Dose 3	JTT-251	One dose of study drug by mouth daily for 24 weeks
JTT-251 Dose 1	JTT-251	One dose of study drug by mouth daily for 24 weeks
Placebo	Placebo	One dose of study drug by mouth daily for 24 weeks

Primary Outcome Measures

Name	Time	Method
Change in six-minute walk distance (6MWD) compared to baseline	24 Weeks
Change in pulmonary vascular resistance (PVR) compared to baseline	4, 12, 24 and 28 Weeks	Assessed by right heart catheterization (RHC)
Change in World Health Organization (WHO) functional classification compared to baseline	24 Weeks

Secondary Outcome Measures

Name	Time	Method
Number of adverse events	28 Weeks
JTT-251 trough plasma concentrations	4, 12 and 24 Weeks