NCT01474109

Macitentan for the Treatment of Digital Ulcers in Systemic Sclerosis Patients

Prospective, Randomized, Placebo-controlled, Double-blind, Multicenter, Parallel Group Study to Assess the Efficacy, Safety and Tolerability of Macitentan in Patients With Ischemic Digital Ulcers Associated With Systemic Sclerosis

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 289 (actual)
• Sponsor: Actelion · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The DUAL-1 study is designed as a multicenter, double-blind two-period study with an initial fixed 16-week Period 1, followed by a Period 2 of variable duration. All patients completing Period 1 will continue on their original randomized treatment into Period 2, until the last randomized patient has completed Period 1. Patients will be randomized in a 1:1:1 ratio (macitentan 3mg: macitentan 10mg: placebo). The primary objective is to demonstrate the effect of macitentan on the reduction of the number of new digital ulcers in patients with systemic sclerosis and ongoing digital ulcers. Other objectives include: * the evaluation of the efficacy of macitentan on hand functionality and DU burden at Week 16 in SSc patients with ongoing DU disease. * the evaluation of the safety and tolerability of macitentan in these patients. * the evaluation of the efficacy of macitentan on time to first DU complication during the entire treatment period.

Detailed description

Recurrent digital ulcers (DU) are a manifestation of vascular disease in patients with systemic sclerosis (SSc), are an important source of morbidity and lead to impaired function in these patients. In this study, we are investigating whether treatment with the endothelin receptor antagonist, macitentan, decreases the development of new digital ulcers in patients with SSc. Macitentan is a highly potent, tissue-targeting dual endothelin receptor antagonist. Through complete blockade of endothelin action, macitentan is expected to protect tissue from the damaging effect of elevated endothelin. This therapy is not approved for the treatment of systemic sclerosis, but the use of an ERA is an attractive approach in combating the structural vascular damage observed in SSc leading to complications such as DUs.

Conditions

• Systemic Sclerosis
• Ulcers

Interventions

Type	Name	Description
DRUG	macitentan 3mg	macitentan 3mg tablet once daily
DRUG	macitentan 10mg	macitentan 10mg tablet once daily
DRUG	placebo	matching placebo once daily

Timeline

• Start date: 2011-12-01
• Primary completion: 2013-11-01
• Completion: 2013-11-01
• First posted: 2011-11-18
• Last updated: 2015-01-06
• Results posted: 2015-01-06

Locations

77 sites across 18 countries: United States, Australia, Belarus, Bulgaria, Canada, Chile, Colombia, Croatia, Czechia, Denmark, Finland, Germany, Hungary, India, Italy, Poland, Russia, Ukraine