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

Tracking Information
First Submitted Date ICMJE	August 23, 2013
First Posted Date ICMJE	August 28, 2013
Last Update Posted Date	April 3, 2018
Actual Study Start Date ICMJE	December 18, 2013
Estimated Primary Completion Date	January 31, 2019 (Final data collection date for primary outcome measure)
Current Primary Outcome Measures ICMJE; (submitted: August 23, 2013)	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 Measures ICMJE	Same as current
Change History	Complete list of historical versions of study NCT01930175 on ClinicalTrials.gov Archive Site
Current Secondary Outcome Measures ICMJE; (submitted: August 23, 2013)	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 Measures ICMJE	Same as current
Current Other Outcome Measures ICMJE	Not Provided
Original Other Outcome Measures ICMJE	Not Provided
Descriptive Information
Brief Title ICMJE	Study of Efficacy and Safety of VAY736 in Patients With Pemphigus Vulgaris
Official Title ICMJE	A Randomized, Partial-blind, Placebo-controlled Trial Evaluating the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of VAY736 in the Treatment of Patients With Pemphigus Vulgaris
Brief Summary	To evaluate the safety, tolerability and efficacy of a single intravneous infusion of VAY736, compared to placebo, in pemphigus vulgaris patients.
Detailed Description	Not Provided
Study Type ICMJE	Interventional
Study Phase	Phase 2
Study Design ICMJE	Allocation: Randomized; Intervention Model: Parallel Assignment; Masking: Double (Participant, Investigator); Primary Purpose: Treatment
Condition ICMJE	Pemphigus Vulgaris
Intervention ICMJE	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.
Study Arms	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
Publications *	Not Provided
* Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
Recruitment Information
Recruitment Status ICMJE	Active, not recruiting
Actual Enrollment ICMJE; (submitted: July 10, 2017)	16
Original Estimated Enrollment ICMJE; (submitted: August 23, 2013)	30
Estimated Study Completion Date	January 31, 2019
Estimated Primary Completion Date	January 31, 2019 (Final data collection date for primary outcome measure)
Eligibility Criteria ICMJE	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.
Sex/Gender	Sexes Eligible for Study: All
Ages	20 Years to 70 Years (Adult, Senior)
Accepts Healthy Volunteers	No
Contacts ICMJE	Contact information is only displayed when the study is recruiting subjects
Listed Location Countries ICMJE	Austria, Bulgaria, Taiwan, United States
Removed Location Countries	Germany, Israel, Italy, United Kingdom
Administrative Information
NCT Number ICMJE	NCT01930175
Other Study ID Numbers ICMJE	CVAY736X2203
Has Data Monitoring Committee	Yes
U.S. FDA-regulated Product	Studies a U.S. FDA-regulated Drug Product: Yes Studies a U.S. FDA-regulated Device Product: No
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
Responsible Party	Novartis ( Novartis Pharmaceuticals )
Study Sponsor ICMJE	Novartis Pharmaceuticals
Collaborators ICMJE	Not Provided
Investigators ICMJE	Study Director: Novartis Pharmaceuticals Novartis Pharmaceuticals
PRS Account	Novartis
Verification Date	April 2018
ICMJE Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP