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

NCT ID: NCT01688882

Last Updated: 2016-04-28

Study Results

Results available

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Basic Information

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Recruitment Status

TERMINATED

Clinical Phase

PHASE2

Total Enrollment

20 participants

Study Classification

INTERVENTIONAL

Study Start Date

2013-01-31

Study Completion Date

2015-03-31

Brief Summary

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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

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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

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Bullous Pemphigoid

Study Design

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Allocation Method

RANDOMIZED

Intervention Model

PARALLEL

Primary Study Purpose

TREATMENT

Blinding Strategy

QUADRUPLE

Participants Caregivers Investigators Outcome Assessors

Study Groups

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QGE031

QGE031 240 mg Q2W s.c.

Group Type EXPERIMENTAL

QGE031

Intervention Type DRUG

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

Placebo

Placebo to Match Q2W s.c.

Group Type PLACEBO_COMPARATOR

Placebo

Intervention Type DRUG

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

Open Label QGE031

Open Label QGE031 Q2W s.c.

Group Type EXPERIMENTAL

QGE031

Intervention Type DRUG

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

Interventions

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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.

Intervention Type DRUG

Placebo

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

Intervention Type DRUG

Eligibility Criteria

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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
Minimum Eligible Age

20 Years

Maximum Eligible Age

80 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Novartis Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Novartis Pharmaceuticals

Role: STUDY_DIRECTOR

Novartis Pharmaceuticals

Locations

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Novartis Investigative Site

Iowa City, Iowa, United States

Site Status

Novartis Investigative Site

Durham, North Carolina, United States

Site Status

Novartis Investigative Site

Vienna, , Austria

Site Status

Novartis Investigative Site

Rouen, Cedex, France

Site Status

Novartis Investigative Site

Dresden, , Germany

Site Status

Novartis Investigative Site

Freiburg im Breisgau, , Germany

Site Status

Novartis Investigative Site

Marburg, , Germany

Site Status

Novartis Investigative Site

Shinjuku-ku, Tokyo, Japan

Site Status

Novartis Investigative Site

Taipei, Taiwan, Taiwan

Site Status

Countries

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United States Austria France Germany Japan Taiwan

Other Identifiers

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CQGE031X2202

Identifier Type: -

Identifier Source: org_study_id

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