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

Phase 2TerminatedNCT01688882

Novartis Pharmaceuticals20 enrolled

Overview

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.

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)

Study Type

INTERVENTIONAL

Allocation

RANDOMIZED

Purpose

TREATMENT

Masking

QUADRUPLE

Enrollment

Conditions

Bullous Pemphigoid

Interventions

QGE031DRUG

QGE031 will be evaluated at various dose levels and regimens, based on the impact on disease of the next highest dose level and regimen.

PlaceboDRUG

Placebo will be used to control for normal variability in disease severity.

Eligibility

Sex: ALLMin age: 20 YearsMax age: 80 Years

Medical Language ↔ Plain English

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

Locations (9)

Novartis Investigative Site

Iowa City, Iowa, United States

Novartis Investigative Site

Durham, North Carolina, United States

Novartis Investigative Site

Vienna, Austria

Novartis Investigative Site

Rouen, Cedex, France

Outcomes

Primary Outcomes

Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12

Clinical Global Assessment of Change (CGA-C) responder rate was the responder rate at 12 weeks based on the CGA-C in bullous pemphigoid (BP). A patient with a CGA-C score of 3 or 4 indicating 'at least marked improvement from baseline' at 12 weeks was considered a responder. The CGA-C is an investigator assessment of change from baseline and is scored as follows: -4 = Very marked worsening (100% worsening); -3 = Marked worsening (67-99% worsening); -2 = Moderate worsening (34-66% worsening); -1 = Slight worsening (1-33% worsening); 1= Slight improvement (1-33% improvement); 2 = Moderate improvement (34-66% improvement); 3 = Marked improvement (67-99% improvement); 4 = Complete clearance (100% improvement)

Time frame: 12 weeks

Secondary Outcomes

Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks

Clinical Global Assessment of Change (CGA-C) responder rate was the responder rate at 6 weeks based on the CGA-C score in bullous pemphigoid (BP). A patient with a CGA-C score of 3 or 4 indicating marked improvement from baseline at 6 weeks was considered a responder. The CGA-C is an investigator assessment of change from baseline and is scored as follows: -4 = Very marked worsening (100% worsening); -3 = Marked worsening (67-99% worsening); -2 = Moderate worsening (34-66% worsening); -1 = Slight worsening (1-33% worsening); 1= Slight improvement (1-33% improvement); 2 = Moderate improvement (34-66% improvement); 3 = Marked improvement (67-99% improvement); 4 = Complete clearance (100% improvement)

Time frame: 6 weeks

Number of Patients Investigator Global Assessment Score Over 12 Weeks

Investigator's Global Assessment (IGA) - (scale of 0 to 4, where 0=clear, 1=almost clear, 2=mild, 3=moderate and 4=severe)

Time frame: Baseline (week 0), week 6 and week 12

Data from ClinicalTrials.gov

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