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

This study is currently recruiting participants. (see Contacts and Locations)
Verified May 2014 by Novartis
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01930175
First received: August 23, 2013
Last updated: May 27, 2014
Last verified: May 2014
  Purpose

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


Condition Intervention Phase
Pemphigus Vulgaris
Drug: VAY736
Drug: Placebo to VAY736
Phase 2

Study Type: Interventional
Study Design: Allocation: Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Investigator)
Primary Purpose: Treatment
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 Vulgaris

Resource links provided by NLM:


Further study details as provided by Novartis:

Primary Outcome Measures:
  • 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.


Secondary Outcome Measures:
  • 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.


Estimated Enrollment: 30
Study Start Date: December 2013
Estimated Study Completion Date: July 2017
Estimated Primary Completion Date: July 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
matching placebo (infusion bag) to VAY736, a single dose administered intravenously over 2 hours.
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.
Experimental: VAY736 Dose 1
Intravenous infusion of VAY736 at Dose Level 1, a single dose administered intravenously for 2 hours
Drug: VAY736
VAY736 will be administered to patients with mild-moderate pemphigus vulgaris in a randomized manner.
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.
Drug: VAY736
VAY736 will be administered to patients with mild-moderate pemphigus vulgaris in a randomized manner, following a safety review.

  Eligibility

Ages Eligible for Study:   20 Years to 70 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT01930175

Contacts
Contact: Novartis Pharmaceuticals 1-888-669-6682
Contact: Novartis Pharmaceuticals

Locations
United States, Iowa
Novartis Investigative Site Not yet recruiting
Iowa City, Iowa, United States, 52242-1091
United States, North Carolina
Novartis Investigative Site Not yet recruiting
Chapel Hill, North Carolina, United States, 27516
United States, Pennsylvania
Novartis Investigative Site Recruiting
Philadelphia, Pennsylvania, United States, 19104
Austria
Novartis Investigative Site Recruiting
Vienna, Austria
Bulgaria
Novartis Investigative Site Not yet recruiting
Sofia, Bulgaria, 1431
Germany
Novartis Investigative Site Withdrawn
Dresden, Germany, 01307
Novartis Investigative Site Withdrawn
Freiburg, Germany, 79104
Novartis Investigative Site Withdrawn
Marburg, Germany, 35039
Israel
Novartis Investigative Site Not yet recruiting
Tel-Aviv, Israel, 64239
Taiwan
Novartis Investigative Site Not yet recruiting
Taipei, Taiwan, 10002
United Kingdom
Novartis Investigative Site Not yet recruiting
London, United Kingdom, SE1 9RT
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

No publications provided

Responsible Party: Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier: NCT01930175     History of Changes
Other Study ID Numbers: CVAY736X2203
Study First Received: August 23, 2013
Last Updated: May 27, 2014
Health Authority: United States: Food and Drug Administration
Germany: Paul-Ehrlich-Institut
United Kingdom: Medicines and Healthcare Products Regulatory Agency
Austria: Agency for Health and Food Safety
Taiwan: Center for Drug Evaluation

Keywords provided by Novartis:
Pemphigus vulgaris
pemphigus
blistering disease

Additional relevant MeSH terms:
Pemphigus
Autoimmune Diseases
Immune System Diseases
Skin Diseases
Skin Diseases, Vesiculobullous

ClinicalTrials.gov processed this record on October 21, 2014