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

Study Status: Terminated
Recruit Status: Terminated
Study Type: Interventional

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

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 Summary:

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)

Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Number of Patients That Had a Clinical Global Assessment of Change (CGA-C) Responder Rate by Week 12 [ Time Frame: 12 weeks ]

Original Primary Outcome: Change in the Clinical Global Assessment of Change from Baseline to Week 12 [ Time Frame: 12 weeks ]

Current Secondary Outcome:

• 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:

• 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 ]

Information By: Novartis

Dates:
Date Received: September 17, 2012
Date Started: January 2013
Date Completion:
Last Updated: March 28, 2016
Last Verified: March 2016