NCT03626688

A Study Evaluating the Efficacy and Safety of Ralinepag to Improve Treatment Outcomes in PAH Patients

A Phase 3, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Ralinepag When Added to PAH Standard of Care or PAH Specific Background Therapy in Subjects With WHO Group 1 PAH

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 687 (actual)
• Sponsor: United Therapeutics · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

Study ROR-PH-301, ADVANCE OUTCOMES, is designed to assess the efficacy and safety of ralinepag when added to pulmonary arterial hypertension (PAH) standard of care or PAH-specific background therapy in subjects with World Health Organization (WHO) Group 1 PAH.

Detailed description

Study ROR-PH-301 is a multicenter, randomized, double-blind, placebo-controlled study. Subjects who meet entry criteria will be randomly allocated 1:1 to receive ralinepag or placebo, in addition to their standard of care or PAH-specific background therapy, as applicable. The primary endpoint is the time (in days) from randomization to the first adjudicated protocol-defined clinical worsening event. All primary endpoint events will be adjudicated by an independent Clinical Event Committee (CEC) in a blinded fashion. Subjects who have a confirmed primary endpoint event adjudicated by the CEC at any time during the study and all subjects on treatment at the conclusion of the study who have completed the Week 28 Visit (after the target number of confirmed events is achieved) will have the option to enroll in an open-label extension (OLE) study. Subjects who do not choose to participate in the OLE study will discontinue study drug and should remain in the study for long-term follow-up of survival status and will receive standard of care PAH treatment, at the discretion of the treating physician.

Conditions

• PAH
• Pulmonary Hypertension
• Pulmonary Arterial Hypertension
• Hypertension
• Connective Tissue Diseases
• Familial Primary Pulmonary Hypertension
• Vascular Diseases
• Cardiovascular Diseases
• Hypertension, Pulmonary
• Lung Diseases
• Respiratory Tract Disease

Interventions

Type	Name	Description
DRUG	Ralinepag	Active
DRUG	Placebo	Placebo

Timeline

• Start date: 2018-08-30
• Primary completion: 2025-12-31
• Completion: 2025-12-31
• First posted: 2018-08-13
• Last updated: 2026-04-14

Locations

209 sites across 33 countries: United States, Argentina, Australia, Austria, Belgium, Brazil, Bulgaria, Canada, Chile, China, Croatia, Czechia, Denmark, France, Germany, Greece, Hungary, Israel, Italy, Mexico, Netherlands, Poland, Portugal, Romania, Serbia, Singapore, South Korea, Spain, Sweden, Taiwan, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study