NCT01688882

Safety, Efficacy and PK/PD of QGE031 vs. Placebo in Patients With Active Bullous Pemphigoid Despite Oral Steroid Treatment

A Randomized, Double-blind, Placebo Controlled, Parallel Group Study Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of QGE031 in the Treatment of Patients With Bullous Pemphigoid With Disease Refractory to Oral Steroid Treatment

• Status: Terminated
• Phase: Phase 2
• Study type: Interventional
• Enrollment: 20 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 20 Years – 80 Years
• Healthy volunteers: Not accepted

Summary

To evaluate the safety and efficacy of QGE031 versus placebo in patients with bullous pemphigoid. Efficacy will be assessed as a reduction of disease activity. How QGE031 is broken down by the body and the impact it has on different blood and tissue markers will also be explored.

Detailed description

This study was planned to be divided into 2 distinct parts. Part 1 was a multicenter, randomized, placebo-controlled study evaluating the efficacy, safety, PK and PD of multiple, subcutaneous doses of QGE031 in the treatment of patients with BP with disease refractory to oral steroid treatment. Patients were treated with QGE031 or placebo in a 2:1 ratio. Part 2 of this study was planned to be a multi-center, open label, dose range finding study evaluating the efficacy, safety, PK and PD of multiple, subcutaneous doses of QGE031 in the treatment of patients with BP with disease refractory to oral steroid treatment. This study was stopped after Part 1 completed and was terminated because the predefined criteria of efficacy was not reached ( \>50% better then placebo)

Conditions

• Bullous Pemphigoid

Interventions

Type	Name	Description
DRUG	QGE031	QGE031 will be evaluated at various dose levels and regimens, based on the impact on disease of the next highest dose level and regimen.
DRUG	Placebo	Placebo will be used to control for normal variability in disease severity.

Timeline

• Start date: 2013-01-01
• Primary completion: 2015-03-01
• Completion: 2015-03-01
• First posted: 2012-09-20
• Last updated: 2016-04-28
• Results posted: 2016-04-28

Locations

9 sites across 6 countries: United States, Austria, France, Germany, Japan, Taiwan