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

September 17, 2012
March 28, 2016
January 2013
March 2015   (final data collection date for primary outcome measure)
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)

Change in the Clinical Global Assessment of Change from Baseline to Week 12 [ Time Frame: 12 weeks ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT01688882 on ClinicalTrials.gov Archive Site
  • 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)
  • 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 ]
Not Provided
Not Provided
 
Safety, Efficacy and PK/PD of QGE031 vs. Placebo in Patients With Active Bullous Pemphigoid Despite Oral Steroid Treatment
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
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.

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)

Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Bullous Pemphigoid
  • 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.
  • 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
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Terminated
20
March 2015
March 2015   (final data collection date for primary outcome measure)

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

Both
20 Years to 80 Years
No
Contact information is only displayed when the study is recruiting subjects
United States,   Austria,   France,   Germany,   Japan,   Taiwan
 
NCT01688882
CQGE031X2202
Yes
Not Provided
Not Provided
Novartis Pharmaceuticals
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
March 2016

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP