Clinical Trial: A Study to Evaluate the Efficacy and Safety of Rituximab Versus Mycophenolate Mofetil (MMF) in Participants With Pemphigus Vulgaris (PV)

Study Status: Recruiting
Recruit Status: Recruiting
Study Type: Interventional

Official Title: A Randomized, Double-Blind, Double-Dummy, Active-Comparator, Multicenter Study to Evaluate the Efficacy and Safety of Rituximab Versus MMF in Patients With Pemphigus Vulgaris

Brief Summary:

This is a Phase III, randomized, double-blind, double-dummy, active-comparator, parallel-arm, multicenter study to evaluate the efficacy and safety of rituximab compared with MMF in participants with moderate-to-severely active PV requiring 60-120 milligrams per day (mg/day) oral prednisone or equivalent. Participants must have a confirmed diagnosis of PV within the previous 24 months (by skin or mucosal biopsy and immunohistochemistry) and evidence of active disease at screening.

Approximately 124 participants will be enrolled at up to 60 centers worldwide. Participants will be randomized in a 1:1 ratio to receive either rituximab plus MMF placebo or rituximab placebo plus MMF. Randomization will be stratified by duration of illness.

The study will consist of three periods: a screening period of up to 28 days, a 52-week double-blind treatment period, and a 48-week safety follow up period that begins at the time of study treatment completion or discontinuation.


Detailed Summary:
Sponsor: Hoffmann-La Roche

Current Primary Outcome: Percentage of Participants Who Achieve Sustained Complete Remission, Evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score [ Time Frame: From baseline up to 52 weeks ]

Original Primary Outcome: Proportion of pts who achieve a sustained complete remission (wound healing with no new active lesions on 0 mg prednisone [equivalent]/day and maintaining response >/=16 consecutive weeks) without experiencing an event that constitutes treatment failure [ Time Frame: 52 weeks ]

Current Secondary Outcome:

  • Time to Protocol Defined Disease Flare [ Time Frame: From baseline up to 52 weeks ]
  • Cumulative Oral Corticosteroid Dose [ Time Frame: From baseline up to 52 weeks ]
  • Duration of Sustained Complete Remission, Evaluated by the PDAI Activity Score [ Time Frame: From baseline up to 52 weeks ]
  • Protocol Defined Disease Flares [ Time Frame: From baseline up to 52 weeks ]
  • Change in Health-Related Quality of Life (HRQoL), as Measured by the Dermatology Life Quality Index (DLQI) Score [ Time Frame: Baseline, Weeks 12, 24, 40, and 52 ]
  • Time to Initial Sustained Complete Remission, Evaluated by the PDAI Activity Score [ Time Frame: From baseline up to 52 weeks ]
  • Change in Participants Impression of PV Symptoms, as Measured by the Patients' Global Impression of Change (PGIC) Questionnaire Score [ Time Frame: Baseline, Weeks 12, 24, 40, and 52 ]
  • Change in Clinician Impression of Participant's PV Symptoms, as Measured by the Clinician Global Impression of Change (CGIC) Questionnaire Score [ Time Frame: Baseline, Weeks 12, 24, 40, and 52 ]
  • Percentage of Participants With Adverse Events, Serious Adverse Events, and Corticosteroid-Related Adverse Events [ Time Frame: Baseline up to 100 weeks ]
  • Percentage of Participants With Human Anti-Chimeric Antibody (HACA) [ Time Frame: Baseline; Weeks 24 and 52/early withdrawal; 48 weeks after end of treatment (end of treatment: up to Week 52) ]
  • Blood Lymphocyte Levels [ Time Frame: Baseline; Weeks 2, 4, 8, 16, 24, 26, 40 and 52; 12, 24, 36, 48 weeks after end of treatment (end of treatment: up to Week 52) ]
  • Plasma Immunoglobulin (Ig) Levels [ Time Frame: Baseline; Weeks 16, 24, 40 and 52; 12, 24, 36, 48 weeks after end of treatment (end of treatment: up to Week 52) ]


Original Secondary Outcome:

  • Change in patients' impression of PV symptoms as measured by the Patients' Global Impression of Change (PGIC) score [ Time Frame: From baseline to Week 52 ]
  • Change in clinician impression of patients' PV symptoms, as measured by the Clinician Global Impression of Change (CGIC) score [ Time Frame: From baseline to Week 52 ]
  • Time to flare (relapse) [ Time Frame: 52 weeks ]
  • Cumulative oral corticosteroid dose over the treatment period [ Time Frame: 52 weeks ]
  • Duration of sustained complete remission [ Time Frame: 52 weeks ]
  • Total number of disease flares during the treatment period [ Time Frame: 52 weeks ]
  • Change in Health-Related Quality of Life (HRQoL), as Measured by the Dermatology Life Quality Index (DLQI) Score [ Time Frame: From baseline to Week 52 ]
  • Time to initial sustained complete remission [ Time Frame: 52 weeks ]


Information By: Hoffmann-La Roche

Dates:
Date Received: March 4, 2015
Date Started: May 31, 2015
Date Completion: September 7, 2019
Last Updated: April 24, 2017
Last Verified: April 2017