Clinical Trial: Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris

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

Official Title: A Randomized, Partial-blind, Placebo-controlled Trial Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of VAY736 in the Treatment of Patients With Pemphigus

Brief Summary: To evaluate the safety, tolerability and efficacy of a single intravneous infusion of VAY736, compared to placebo, in pemphigus vulgaris patients.

Detailed Summary:
Sponsor: Novartis Pharmaceuticals

Current Primary Outcome: Efficacy of a single intravenous dose of VAY736 in reducing the clinical disease activity in pemphigus vulgaris patinets. [ Time Frame: baseline, 12 weeks ]

The effect of VAY736 on clinical disease activity will be measured by the change in Pemphigus Disease Area Index (PDAI) between baseline and week 12. PDAI is an assessment of skin and mucosal disease activity by the Investigator. The score incorporates the number and size of lesions across multiple skin locations and the mucous membranes.


Original Primary Outcome: Same as current

Current Secondary Outcome:

  • Number of patients with adverse events [ Time Frame: up to an average of 12 months ]
    Summarized statistics on adverse events will be reported under categories such as total adverse events, serious adverse events and death
  • Change in Autoimmune Skin disease Intensity Score (ABSIS) [ Time Frame: 12 weeks ]
    The effect of VAY736 on the ABSIS score will be compared betwen baseline and Week 12. ABSIS is a quality and quantity-based score of skin and oral mucosal lesions, combining the extent of the affected body surface area, the quality of the lesion and the extent of oral mucosal involvement, including food-related pain and discomfort.
  • Change in Investigator Global Assessment (IGA) [ Time Frame: baseline, 12 weeks ]
    The effect of VAY736 on the IGA of a patient's disease activity will compared between baseline and Week 12. The IGA score ranges from 0 (no sign of active disease) to 4 (Severe active disease)
  • VAY736 serum concentration [ Time Frame: 0, 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks ]
    The concentration of VAY736 will be measured in the serum. Descriptive statistics will include area under the concentration curve, half-life and time to maximum concentration.


Original Secondary Outcome: Same as current

Information By: Novartis

Dates:
Date Received: August 23, 2013
Date Started: December 18, 2013
Date Completion: February 13, 2018
Last Updated: May 15, 2017
Last Verified: May 2017