Pemphigus vulgaris (PV) is a rare, chronic, debilitating, and potentially life-threatening autoimmune disorder that is characterized by mucocutaneous blisters.Abatacept is a biologic drug that belongs to the class of T-cell co-stimulation modulators and is used for the treatment of autoimmune diseases.
The background therapy is based on prednisolone administration. PV is a rare disorder, therefore this study is designed as a crossover that may require fewer patients than a parallel study. The study enrolled participants with moderate-to-severely active PV requiring ≥ 50 milligrams per day (mg/day) oral prednisone or equivalent. The purpose of this study was to evaluate the efficacy, tolerability, and safety of abatacept injection for subcutaneous use (abatacept SC) 150 mg administered once in a week in subjects with PV. It was anticipated that with sustained immune suppression in the presence of abatacept SC that clinical remission of the disease would be improved.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
DOUBLE
Enrollment
60
Abatacept (Orencia) was provided in prefilled glass syringes initial syringes contained 0.6ml (60mg) of concentration 100 mg/m: drug product subsequently modified to 0.4 mL (20mg) concentration (50mg/ML) drug product. Combined with standard of care prednisone 10-40mg qd
MMF will be administered at a starting dose of 1000 milligrams (mg) Q12H and the dose will be tapered to achieve a goal of 0.5-1.0 gram (gm) Q12H. Combined with standard of care prednisone 10-40mg qd through 52 weeks.
Tongji Hospital
Wuhan, Hubei, China
RECRUITINGPercentage of Participants Who Achieved Sustained Complete Remission, Evaluated by the Pemphigus Disease Area Index (PDAI) Activity Score
PADAI and ABSIS was proposed by the German Blistering Disease Group in 2007 which was accepted as the most sensitive and reliable systems for evaluation of pemphigus severity. PADAI was developed by the International Pemphigus Definitions Group in 2009
Time frame: 24 Weeks
Percentage of Participants Who Achieved Sustained Complete Remission, Evaluated byAutoimmune bullous skin disorder intensity score (ABSIS)
PADAI and ABSIS was proposed by the German Blistering Disease Group in 2007 which was accepted as the most sensitive and reliable systems for evaluation of pemphigus severity. PADAI was developed by the International Pemphigus Definitions Group in 2009
Time frame: 24 Weeks
Cumulative Oral Corticosteroid Dose
Calculate the Cumulative Oral Corticosteroid Dose during 52 week
Time frame: From 12th, 24th, 36th and 52th Week
Ulcer Severity Score (USS) for the assessment of skin, oral ulcer improvement
The USS incorporates six ulcer characteristics: number, size, duration, ulcer-free period, site, and pain. This scoring template may be of value to future studies assessing treatment efficacy.
Time frame: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Physician global assessment (PGA)
Physician global assessment was assessed by an individual researcher
Time frame: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Autoimmune bullous disease quality of life (ABQoL)
Total ABQoL scores range from 0 to 30 with higher DLQI scores reflecting greater impairment in a participant's health-related quality of life. The ABQoL score is calculated by summing the score of each question resulting in a maximum of 30 and a minimum of 0. The higher the score, the more quality of life is impaired. The measure type mean is the estimated mean from adjusted MMRM.
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Time frame: From baseline up to 4th, 8th,12th, 24th, 36th and 52th Week
Anti-desmoglein 1 (anti-Dsg1) and anti-Dsg3 autoantibody titers
anti-Dsg1 and anti-Dsg3 will be performed using ELISA
Time frame: From Baseline up to 12th, 24th and 52th Week
Change From Baseline for CD19+ B Cell Count
CD19+ B cell count will be performed using Flow Cytometry
Time frame: From Baseline up to 12th, 24th and 52th Week