Clinical trial NCT01688882: Safety, Efficacy and PK/PD of QGE031 vs. Placebo in Patients With Active Bullous Pemphigoid Despite Oral Steroid Treatment
Official title: 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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE2
Conditions: Bullous Pemphigoid
Keywords: bullous pemphigoid
QGE031
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2012-09-20
Enrollment: 20
Sex: ALL
Minimum age (years): 20
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

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)

Eligibility criteria: 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

Primary outcomes:
measure: Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12
timeFrame: 12 weeks
Secondary outcomes:
measure: Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks
timeFrame: 6 weeks
measure: Number of Patients Investigator Global Assessment Score Over 12 Weeks
timeFrame: Baseline (week 0), week 6 and week 12
Other outcomes:
Locations:
city: Iowa City
country: United States
facility: Novartis Investigative Site
state: Iowa
city: Durham
country: United States
facility: Novartis Investigative Site
state: North Carolina
city: Vienna
country: Austria
facility: Novartis Investigative Site
city: Rouen
country: France
facility: Novartis Investigative Site
state: Cedex
city: Dresden
country: Germany
facility: Novartis Investigative Site
city: Freiburg
country: Germany
facility: Novartis Investigative Site
city: Marburg
country: Germany
facility: Novartis Investigative Site
city: Shinjuku-ku
country: Japan
facility: Novartis Investigative Site
state: Tokyo
city: Taipei
country: Taiwan
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: