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

Novartis

Status and phase: Terminated; Phase 2

Conditions: Bullous Pemphigoid

Treatments: Drug: QGE031; Drug: Placebo

Study type: Interventional

Funder types: Industry

Identifiers

NCT01688882

CQGE031X2202

Details and patient eligibility

About

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.

Full 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)

Enrollment: 20 patients

Sex: All

Ages: 20 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Patients diagnosed with bullous pemphigoid
• Stable dose of prednisone at or above 10mg per day but no greater than 1 mg/kg/day
• Weigh between 40-120kg
• total IgE level up to 5000 IU/mL

Exclusion criteria

• Use of rituximab within 1 year

Other protocol-defined inclusion/exclusion criteria may apply

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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20 participants in 3 patient groups, including a placebo group

QGE031

Experimental group

Description:

QGE031 240 mg Q2W s.c.

Treatment:

Drug: QGE031

Placebo

Placebo Comparator group

Description:

Placebo to Match Q2W s.c.

Treatment:

Drug: Placebo

Open Label QGE031

Experimental group

Description:

Open Label QGE031 Q2W s.c.

Treatment:

Drug: QGE031