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

• 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 ]

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)

• 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

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