Conditions: Bullous Pemphigoid
Keywords: bullous pemphigoid QGE031
Allocation: RANDOMIZED | Intervention model: PARALLEL
Enrollment: 20
Healthy volunteers: no

Brief 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)

Arms:
arm 1: QGE031 240 mg Q2W s.c. arm 2: Placebo to Match Q2W s.c. arm 3: Open Label QGE031 Q2W s.c.

Interventions: intervention 1: QGE031 intervention 2: Placebo
Intervention descriptions: intervention 1: QGE031 will be evaluated at various dose levels and regimens, based on the impact on disease of the next highest dose level and regimen. intervention 2: Placebo will be used to control for normal variability in disease severity.

Protocol document: