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Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris

This study is currently recruiting participants. (see Contacts and Locations)
Verified November 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01930175
First received: August 23, 2013
Last updated: November 3, 2014
Last verified: November 2014

August 23, 2013
November 3, 2014
December 2013
July 2017   (final data collection date for primary outcome measure)
Efficacy of a single intravenous dose of VAY736 in reducing the clinical disease activity in pemphigus vulgaris patinets. [ Time Frame: baseline, 12 weeks ] [ Designated as safety issue: No ]
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.
Efficacy of a single intravenous dose of VAY736 in reducing the clinical disease activity in pemphigus vulgaris patinets. [ Time Frame: baseline, 12 weeks ] [ Designated as safety issue: No ]
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.
Complete list of historical versions of study NCT01930175 on ClinicalTrials.gov Archive Site
  • Number of patients with adverse events [ Time Frame: up to an average of 12 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.
  • Number of patients with adverse events [ Time Frame: up to an average of 12 months ] [ Designated as safety issue: Yes ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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 ] [ Designated as safety issue: No ]
    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.
Not Provided
Not Provided
 
Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris
A Randomized, Partial-blind, Placebo-controlled Trial Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of VAY736 in the Treatment of Patients With Pemphigus Vulgaris

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

Not Provided
Interventional
Phase 2
Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
Pemphigus Vulgaris
  • Drug: VAY736
    VAY736 will be administered to patients with mild-moderate pemphigus vulgaris in a randomized manner.
  • Drug: Placebo to VAY736
    Placebo to VAY736 will be administered to patients with mild-moderate pemphigus vulgaris in a randomized manner. All patients that receive placebo will be offered open-label VAY736.
  • Drug: VAY736
    VAY736 will be administered to patients with mild-moderate pemphigus vulgaris in a randomized manner, following a safety review.
  • Placebo Comparator: Placebo
    matching placebo (infusion bag) to VAY736, a single dose administered intravenously over 2 hours.
    Intervention: Drug: Placebo to VAY736
  • Experimental: VAY736 Dose 1
    Intravenous infusion of VAY736 at Dose Level 1, a single dose administered intravenously for 2 hours
    Intervention: Drug: VAY736
  • Experimental: VAY736 Dose 2
    Intravenous infusion of VAY736 at Dose Level 2, a single dose administered intravenously over 2 hours. Dose level 2 will be initiated following a safety review of patients receiving Dose Level 1 or placebo.
    Intervention: Drug: VAY736
Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruiting
32
July 2017
July 2017   (final data collection date for primary outcome measure)

Inclusion Criteria:

  • Adult patients 20 to 70 years of age
  • Confirmed diagnosis of pemphigus vulgaris
  • Presence of mild to moderate pemphigus vulgaris
  • Patients must weight between 40 kg and 150 kg inclusive
  • on a stable dose of oral corticosteriod therapy (with or without azathioprine or mycophenolate)

Exclusion Criteria:

  • Pregnant or nursing (lactating) women
  • Women of child-bearing potential unless they are using a highly effective method of birth control during dosing and for 4 months following study treatment
  • Recent previous treatment with photo therapy, biological therapy, steroids, immunosuppresive agents (unless washout period applied)
  • Active or recent history of clinically significant infection
  • use of rituximab within 1 year Other protocol-defined inclusion/exclusion criteria may apply.
Both
20 Years to 70 Years
No
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals
United States,   Austria,   Bulgaria,   Germany,   Israel,   Italy,   Taiwan,   United Kingdom
 
NCT01930175
CVAY736X2203
Yes
Novartis ( Novartis Pharmaceuticals )
Novartis Pharmaceuticals
Not Provided
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
Novartis
November 2014

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