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

NCT01930175 | Terminated Phase 2 Results DMC FDA Drug

• 1 other identifier: CVAY736X2203
• Study Type: interventional
• Target: 13
• Locations: 4 countries
• Sites: 5

Brief Summary

The study evaluated the efficacy, safety and pharmacokinetics of VAY736 in the treatment of patients with pemphigus vulagaris (PV).

Conditions

Pemphigus Vulgaris

Keywords

Pemphigus vulgaris; pemphigus; blistering disease

Outcome Measures

Primary Outcomes (1)

• Pemphigus Disease Area Index (PDAI) at Week 12

  PDAI is specific cutaneous and mucosal disease activity assessment performed by investigator based on evaluation of lesions in well-defined anatomical locations. The score weighted for the number and size of lesions with score of 0 (absent) to 10 given for skin (12 body locations), scalp and mucous membrane showing disease activity (erosions/blisters or new erythema). Damage, such as post inflammatory hyperpigmentation or erythema from resolving lesion, scored separately from the main score as absent (0) or present (1) for each body area or scalp resulting in a score of 0 to 12 or 0 to 1, respectively. Thus, PDAI ranged from 0 to 263, with 250 points representing disease activity (120 points for skin activity; 10 points for scalp activity; 120 points for mucosal activity) and 13 points representing disease damage.

  Week 12

Secondary Outcomes (7)

• Autoimmune Skin Disease Intensity Score (ABSIS) at Baseline and Week 12.

  Baseline, Week 12

• Change From Baseline in Investigator Global Assessment (IGA) at Week 12

  Baseline, Week 12

• VAY736 Serum Concentration - AUCinf

  predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks

• VAY736 Serum Concentration - AUClast

  predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks

• VAY736 Serum Concentration - Cmax

  predose, 2, 24 hours and weeks 1, 2, 3, 6, 9, 12, 16, 20, 24 and approximately 52 weeks

Study Arms (3)

Placebo

PLACEBO COMPARATOR

single dose iv of Placebo

Drug: Placebo

VAY736 3 mg/kg

EXPERIMENTAL

single dose iv of VAY736 at a dose of 3mg/kg

Drug: VAY736

VAY736 10 mg/kg

EXPERIMENTAL

single dose iv of VAY736 at a dose of 10mg/kg initiated following a safety review of patients receiving VAY736 3 mg/kg or placebo.

Drug: VAY736

Interventions

VAY736 DRUG

Also known as: Ianalumab

VAY736 10 mg/kg; VAY736 3 mg/kg

Placebo DRUG

Placebo

Eligibility Criteria

• Age: 20 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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)

You may not qualify if:

• 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

Sponsors & Collaborators

• Novartis Pharmaceuticals: lead

Study Sites (5)

Novartis Investigative Site

Chapel Hill, North Carolina, 27516, United States

Location

Novartis Investigative Site

Philadelphia, Pennsylvania, 19104, United States

Location

Novartis Investigative Site

Vienna, 1040, Austria

Location

Novartis Investigative Site

Sofia, BGR, 1431, Bulgaria

Location

Novartis Investigative Site

Taipei, 10002, Taiwan

Location

Related Links

• A Plain Language Trial Summary is available on novartisclinicaltrials.com

MeSH Terms

Conditions

Pemphigus

Interventions

ianalumab

Condition Hierarchy (Ancestors)

Skin Diseases, Vesiculobullous; Skin Diseases; Skin and Connective Tissue Diseases; Autoimmune Diseases; Immune System Diseases

Results Point of Contact

• Title: Study Director
• Organization: Novartis Pharmaceuticals

Novartis.email@novartis.com

862-778-8300

Study Officials

• Novartis Pharmaceuticals

  Novartis Pharmaceuticals

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: OTHER
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 23, 2013
• First Posted: August 28, 2013
• Study Start: December 18, 2013
• Primary Completion: September 25, 2019
• Study Completion: September 25, 2019
• Last Updated: October 8, 2021
• Results First Posted: October 19, 2020

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will share

Locations

US (2); BU (1); AU (1); TW (1)