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

Bullous Pemphigoid Clinical Trial

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

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT01688882
• Other study ID #: CQGE031X2202
• Status: Terminated
• Phase: Phase 2
• First received: September 17, 2012
• Last updated: October 14, 2015
• Start date: January 2013
• Est. completion date: March 2015

Study information

• Verified date: October 2015
• Source: Novartis
• Contact: n/a
• Is FDA regulated: No
• Health authority: United States: Food and Drug AdministrationAustria: Agency for Health and Food SafetyGermany: Paul-Ehrlich-InstitutTaiwan: Center for Drug EvaluationFrance: Ministry of Health
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 20
• Est. completion date: March 2015
• Est. primary completion date: March 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 20 Years to 80 Years

Eligibility

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

Study Design

Allocation: Randomized, Endpoint Classification: Safety/Efficacy Study, Intervention Model: Parallel Assignment, Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor), Primary Purpose: Treatment

Related Conditions & MeSH terms

• Bullous Pemphigoid
• Pemphigoid, Bullous

Intervention

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

Locations

Country	Name	City	State
Austria	Novartis Investigative Site	Vienna
France	Novartis Investigative Site	Rouen	Cedex
Germany	Novartis Investigative Site	Dresden
Germany	Novartis Investigative Site	Freiburg
Germany	Novartis Investigative Site	Marburg
Japan	Novartis Investigative Site	Shinjuku-ku	Tokyo
Taiwan	Novartis Investigative Site	Taipei
United States	Novartis Investigative Site	Durham	North Carolina
United States	Novartis Investigative Site	Iowa City	Iowa

Sponsors (1)

Lead Sponsor	Collaborator
Novartis Pharmaceuticals

Countries where clinical trial is conducted

United States,  Austria,  France,  Germany,  Japan,  Taiwan, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Change in the Clinical Global Assessment of Change from Baseline to Week 12		12 weeks	No
Secondary	Change in the Clinical Global Assessment of Change from Baseline to Week 6		6 weeks	No
Secondary	Change from baseline in the Investigator Global Assessment over 48 weeks		Baseline, every 2 weeks up to 12 weeks, every 4 weeks up to 24 weeks, every 8 weeks up to 48 weeks	No
Secondary	Safety of QGE031 over 48 weeks		Baseline, ever 2 weeks up to 12 weeks, every 4 weeks up to 24 weeks, every 8 weeks up to 48 weeks	Yes