Clinical trial NCT03789643: Study to Evaluate Efficacy and Safety of JTT-251 in Participants With Pulmonary Arterial Hypertension
Official title: A Phase 2, Multicenter, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of JTT-251 Administered for 24 Weeks to Participants With Pulmonary Arterial Hypertension (RELIEF-PAH)
Study type: INTERVENTIONAL
Overall status: WITHDRAWN
Phase: PHASE2
Conditions: Pulmonary Arterial Hypertension
Keywords: JTT-251
Pulmonary arterial hypertension
RELIEF-PAH
Six-minute walk distance (6MWD)
Lead sponsor: Akros Pharma Inc.
Sponsor class: INDUSTRY
First posted: 2018-12-28
Enrollment: 0
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Change in six-minute walk distance (6MWD) compared to baseline
timeFrame: 24 Weeks
measure: Change in World Health Organization (WHO) functional classification compared to baseline
timeFrame: 24 Weeks
measure: Change in pulmonary vascular resistance (PVR) compared to baseline
timeFrame: 4, 12, 24 and 28 Weeks
Secondary outcomes:
measure: Number of adverse events
timeFrame: 28 Weeks
measure: JTT-251 trough plasma concentrations
timeFrame: 4, 12 and 24 Weeks
Other outcomes:
Locations:
Study officials:
References: