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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01688882
First received: September 17, 2012
Last updated: May 21, 2014
Last verified: May 2014
  Purpose

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.


Condition Intervention Phase
Bullous Pemphigoid
Drug: QGE031
Drug: Placebo
Phase 2

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

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • Change in the Clinical Global Assessment of Change from Baseline to Week 12 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Change in the Clinical Global Assessment of Change from Baseline to Week 6 [ Time Frame: 6 weeks ] [ Designated as safety issue: No ]
  • Change from baseline in the Investigator Global Assessment over 48 weeks [ Time Frame: Baseline, every 2 weeks up to 12 weeks, every 4 weeks up to 24 weeks, every 8 weeks up to 48 weeks ] [ Designated as safety issue: No ]
  • Safety of QGE031 over 48 weeks [ Time Frame: Baseline, ever 2 weeks up to 12 weeks, every 4 weeks up to 24 weeks, every 8 weeks up to 48 weeks ] [ Designated as safety issue: Yes ]

Estimated Enrollment: 42
Study Start Date: January 2013
Estimated Study Completion Date: November 2015
Estimated Primary Completion Date: November 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: High dose QGE031 with interval 1 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.
Experimental: High dose QGE031 with interval 2 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.
Experimental: Low dose QGE031 with interval 2 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 Comparator: Placebo Drug: Placebo
Placebo will be used to control for normal variability in disease severity.

  Eligibility

Ages Eligible for Study:   20 Years to 80 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
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

  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01688882

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals

Locations
United States, California
Novartis Investigative Site Recruiting
Los Angeles, California, United States, 90033
United States, Iowa
Novartis Investigative Site Not yet recruiting
Iowa City, Iowa, United States, 52242
United States, North Carolina
Novartis Investigative Site Recruiting
Durham, North Carolina, United States, 27710
United States, Pennsylvania
Novartis Investigative Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
Austria
Novartis Investigative Site Recruiting
Vienna, Austria
France
Novartis Investigative Site Recruiting
Rouen, Cedex, France, 76031
Germany
Novartis Investigative Site Recruiting
Berlin, Germany, 10098
Novartis Investigative Site Recruiting
Dresden, Germany, 01307
Novartis Investigative Site Recruiting
Freiburg, Germany, 79104
Novartis Investigative Site Recruiting
Hannover, Germany, 30449
Novartis Investigative Site Not yet recruiting
Luebeck, Germany, 23538
Novartis Investigative Site Recruiting
Marburg, Germany, 35039
Japan
Novartis Investigative Site Not yet recruiting
Okayama-city, Okayama, Japan, 700-8558
Novartis Investigative Site Recruiting
Bunkyo-ku, Tokyo, Japan, 113-8431
Novartis Investigative Site Recruiting
Shinjuku-ku, Tokyo, Japan, 160-8582
Taiwan
Novartis Investigative Site Not yet recruiting
Niaosong Township, Taiwan, 83301
Novartis Investigative Site Recruiting
Taipei, Taiwan, 10002
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01688882     History of Changes
Other Study ID Numbers: CQGE031X2202
Study First Received: September 17, 2012
Last Updated: May 21, 2014
Health Authority: United States: Food and Drug Administration
Austria: Agency for Health and Food Safety
Germany: Paul-Ehrlich-Institut
Taiwan: Center for Drug Evaluation
France: Ministry of Health

Keywords provided by Novartis:
bullous pemphigoid
QGE031

Additional relevant MeSH terms:
Pemphigoid, Bullous
Skin Diseases, Vesiculobullous
Skin Diseases
Autoimmune Diseases
Immune System Diseases

ClinicalTrials.gov processed this record on September 29, 2014