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

This study has been terminated.
(This study was stopped after Part 1 completed and was terminated because the predefined criteria of efficacy was not reached ( >50% better then placebo))
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01688882
First received: September 17, 2012
Last updated: March 28, 2016
Last verified: March 2016
  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:
  • Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]

    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)



Secondary Outcome Measures:
  • Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks [ Time Frame: 6 weeks ] [ Designated as safety issue: No ]

    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)


  • Number of Patients Investigator Global Assessment Score Over 12 Weeks [ Time Frame: Baseline (week 0), week 6 and week 12 ] [ Designated as safety issue: No ]
    Investigator's Global Assessment (IGA) - (scale of 0 to 4, where 0=clear, 1=almost clear, 2=mild, 3=moderate and 4=severe)


Enrollment: 20
Study Start Date: January 2013
Study Completion Date: March 2015
Primary Completion Date: March 2015 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: QGE031
QGE031 240 mg Q2W s.c.
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
Placebo to Match Q2W s.c.
Drug: Placebo
Placebo will be used to control for normal variability in disease severity.
Experimental: Open Label QGE031
Open Label QGE031 Q2W s.c.
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.

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

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

Locations
United States, Iowa
Novartis Investigative Site
Iowa City, Iowa, United States, 52242
United States, North Carolina
Novartis Investigative Site
Durham, North Carolina, United States, 27710
Austria
Novartis Investigative Site
Vienna, Austria, A 1040
France
Novartis Investigative Site
Rouen, Cedex, France, 76031
Germany
Novartis Investigative Site
Dresden, Germany, 01307
Novartis Investigative Site
Freiburg, Germany, 79104
Novartis Investigative Site
Marburg, Germany, 35039
Japan
Novartis Investigative Site
Shinjuku-ku, Tokyo, Japan, 160-8582
Taiwan
Novartis Investigative Site
Taipei, Taiwan, 10002
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01688882     History of Changes
Other Study ID Numbers: CQGE031X2202 
Study First Received: September 17, 2012
Results First Received: March 28, 2016
Last Updated: March 28, 2016
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
Autoimmune Diseases
Immune System Diseases
Skin Diseases
Skin Diseases, Vesiculobullous

ClinicalTrials.gov processed this record on May 26, 2016