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

Tracking Information
First Submitted Date ICMJE	September 17, 2012
First Posted Date ICMJE	September 20, 2012
Results First Submitted Date	March 28, 2016
Results First Posted Date	April 28, 2016
Last Update Posted Date	April 28, 2016
Study Start Date ICMJE	January 2013
Actual Primary Completion Date	March 2015 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: March 28, 2016)	Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12 [ Time Frame: 12 weeks ]; 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)
Original Primary Outcome Measures ICMJE; (submitted: September 17, 2012)	Change in the Clinical Global Assessment of Change from Baseline to Week 12 [ Time Frame: 12 weeks ]
Change History	Complete list of historical versions of study NCT01688882 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: March 28, 2016)	Response Based on Clinical Global Assessment of Change CGA-C Score at 6 Weeks [ Time Frame: 6 weeks ]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 ]Investigator's Global Assessment (IGA) - (scale of 0 to 4, where 0=clear, 1=almost clear, 2=mild, 3=moderate and 4=severe)
Original Secondary Outcome Measures ICMJE; (submitted: September 17, 2012)	Change in the Clinical Global Assessment of Change from Baseline to Week 6 [ Time Frame: 6 weeks ]; 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 ]; 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 ]
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Safety, Efficacy and PK/PD of QGE031 vs. Placebo in Patients With Active Bullous Pemphigoid Despite Oral Steroid Treatment
Official Title ICMJE	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
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)
Study Type ICMJE	Interventional
Study Phase	Phase 2
Study Design ICMJE	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor); Primary Purpose: Treatment
Condition ICMJE	Bullous Pemphigoid
Intervention ICMJE	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.; Drug: Placebo Placebo will be used to control for normal variability in disease severity.
Study Arms	Experimental: QGE031 QGE031 240 mg Q2W s.c. Intervention: Drug: QGE031; Placebo Comparator: Placebo Placebo to Match Q2W s.c. Intervention: Drug: Placebo; Experimental: Open Label QGE031 Open Label QGE031 Q2W s.c. Intervention: Drug: QGE031
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Terminated
Actual Enrollment ICMJE; (submitted: April 14, 2015)	20
Original Estimated Enrollment ICMJE; (submitted: September 17, 2012)	42
Actual Study Completion Date	March 2015
Actual Primary Completion Date	March 2015 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	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
Sex/Gender	Sexes Eligible for Study: All
Ages	20 Years to 80 Years (Adult, Senior)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	Austria, France, Germany, Japan, Taiwan, United States
Removed Location Countries
Administrative Information
NCT Number ICMJE	NCT01688882
Other Study ID Numbers ICMJE	CQGE031X2202
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Not Provided
IPD Sharing Statement	Not Provided
Responsible Party	Novartis ( Novartis Pharmaceuticals )
Study Sponsor ICMJE	Novartis Pharmaceuticals
Collaborators ICMJE	Not Provided
Investigators ICMJE	Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
PRS Account	Novartis
Verification Date	March 2016
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP