NCT02915835

Riociguat in Scleroderma Associated Digital Ulcers

A Multi-center Randomized, Double Blind, Placebo-controlled, Pilot Study to Assess the Efficacy and Safety of Riociguat in Scleroderma - Associated Digital Ulcers

• Status: Completed
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 17 (actual)
• Sponsor: Dinesh Khanna, MD, MS · Academic / Other
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

The primary objective of this study is to provide preliminary data on the efficacy (digital ulcer net burden) and safety of riociguat administered 3 times daily (TID) in comparison to placebo in patients with scleroderma-associated digital ulcers

Detailed description

This clinical trial is a US, multicenter, double-blind, randomized placebo-controlled, parallel- group study with a total of 20 participants planned to be randomized (approximately 10 participants to the riociguat group and 10 to the placebo group). In addition, a standardized wound care protocol will be followed by the investigators and digital photography will be taken of the cardinal ulcer. The study will allow standard of care medications for the management of DU as background therapy. These may include calcium channel blockers, low dose aspirin, angiotensin enzyme inhibitors, etc. and will be determined by the participant's local physician. The study design consists of three phases: * Screening phase: up to 2 weeks * Double-blind Treatment phase: 16 weeks of double-blind treatment, consisting of: * Dose titration period of up to 8 weeks, and * Stable dosing period of up to 8 weeks * Open-label Extension phase for participants with active DU at the end of the double- blind treatment phase or development of an active DU within a month of completing double-blind phase, consisting of: * Dose titration phase of up to 8 weeks * Stable dosing period for 8 weeks

Conditions

• Scleroderma
• Digital Ulcers

Interventions

Type	Name	Description
DRUG	Riociguat	riociguat 0.5 mg, 1 mg, 1.5 mg, 2 mg and 2.5 mg administered TID; dose titration starting with 1.0 mg (planned up-titration every 2 weeks, with possibility of dose reduction for tolerability; 0.5 mg is the lowest dose and 2.5 mg is the highest dose to be administered)
DRUG	Placebo	Placebo 0.5 mg, 1 mg, 1.5 mg, 2 mg and 2.5 mg administered TID;

Timeline

• Start date: 2016-09-01
• Primary completion: 2018-07-24
• Completion: 2018-07-24
• First posted: 2016-09-27
• Last updated: 2019-09-24
• Results posted: 2019-09-24

Locations

5 sites across 1 country: United States