NCT03055234

Study to Evaluate Efficacy and Safety of Oral Treprostinil in Subjects With Pulmonary Hypertension (PH) Associated With Sickle Cell Disease (SCD)

A Phase 3, Multicenter, Randomized, Placebo-controlled Study to Evaluate Efficacy and Safety of Oral Treprostinil in Subjects With Pulmonary Hypertension Associated With Sickle Cell Disease

• Status: Withdrawn
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 0 (actual)
• Sponsor: United Therapeutics · Industry
• Sex: All
• Age: 12 Years
• Healthy volunteers: Not accepted

Summary

This is a multicenter, randomized (2:1; oral treprostinil:placebo), double-blind, placebo-controlled event-driven (time to pulmonary hypertension \[PH\] clinical worsening) study in subjects with PH associated with sickle cell disease (SCD). Once enrolled, subjects will be evaluated at Weeks 6, 12, 24, and then every 12 weeks for the duration of the study. Subjects will be permitted to enter a 48-week open-label extension period if they experience a PH clinical worsening event.

Conditions

• Pulmonary Hypertension Associated With Sickle Cell Disease

Interventions

Type	Name	Description
DRUG	Oral Treprostinil	Extended-release oral tablet for TID administration
DRUG	Placebo	Matching placebo for TID administration

Timeline

• Start date: 2017-06-01
• Primary completion: 2021-12-01
• Completion: 2022-12-01
• First posted: 2017-02-16
• Last updated: 2017-04-11

Regulatory

• FDA-regulated drug study