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

This study is ongoing, but not recruiting participants.
Sponsor:
Information provided by (Responsible Party):
Novartis ( Novartis Pharmaceuticals )
ClinicalTrials.gov Identifier:
NCT01930175
First received: August 23, 2013
Last updated: July 13, 2017
Last verified: July 2017
  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
Intervention Model: Parallel Assignment
Masking: Participant, 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 ( Novartis Pharmaceuticals ):

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 ]
    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 ]
    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.


Enrollment: 16
Actual Study Start Date: December 18, 2013
Estimated Study Completion Date: February 13, 2018
Estimated Primary Completion Date: February 13, 2018 (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   (Adult, Senior)
Sexes Eligible for Study:   All
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

Locations
United States, North Carolina
Novartis Investigative Site
Chapel Hill, North Carolina, United States, 27516
United States, Pennsylvania
Novartis Investigative Site
Philadelphia, Pennsylvania, United States, 19104
Austria
Novartis Investigative Site
Vienna, Austria, 1040
Bulgaria
Novartis Investigative Site
Sofia, Bulgaria, 1431
Taiwan
Novartis Investigative Site
Taipei, Taiwan, 10002
Sponsors and Collaborators
Novartis Pharmaceuticals
Investigators
Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
  More Information

Responsible Party: Novartis Pharmaceuticals
ClinicalTrials.gov Identifier: NCT01930175     History of Changes
Other Study ID Numbers: CVAY736X2203
Study First Received: August 23, 2013
Last Updated: July 13, 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description:

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations.

This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com


Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No

Keywords provided by Novartis ( Novartis Pharmaceuticals ):
Pemphigus vulgaris
pemphigus
blistering disease

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

ClinicalTrials.gov processed this record on July 26, 2017