This is a prospective, multicenter, randomized, double-blinded, placebo-controlled trial to investigate the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK, and PD of efgartigimod PH20 SC in adult participants aged from 18 years with PV or PF. The trial comprises a screening period of up to 3 weeks, a treatment period of up to 30 weeks, and an 8-week follow-up period for participants who do not enroll into the open-label extension (OLE) trial ARGX-113-1905. The primary objective of the ARGX-113-1904 trial is to demonstrate the efficacy of subcutaneous administration of efgartigimod co-formulated with recombinant human hyaluronidase PH20 (Efgartigimod PH20 SC) compared to placebo in the treatment of participants with Pemphigus Vulgaris (PV). Secondary objectives are to also demonstrate the efficacy of efgartigimod PH20 SC in the treatment of participants with Pemphigus Foliaceus (PF), and to demonstrate early onset of action and a prednisone-sparing effect. After confirmation of eligibility, participants will be randomized in a 2: 1 ratio to receive efgartigimod PH20 SC or placebo
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
222
Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer
Subcutaneous injection of placebo
Oral prednisone tablets
Investigator site 77 - US0010086
Birmingham, Alabama, United States
Investigator site 97 - US0010091
Scottsdale, Arizona, United States
Investigator site 121 - US0010092
Redwood City, California, United States
Investigator site 125 - US0010153
Castle Rock, Colorado, United States
Investigator site 2 - US0010087
Boca Raton, Florida, United States
Number of Pemphigus Vulgaris (PV) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy
Proportion of participants with pemphigus vulgaris who had CRmin within 30 weeks, defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.
Time frame: up to 30 weeks treatment period
Number of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) Participants Who Achieve Complete Clinical Remission (CR) on Minimal Prednisone Therapy Within 30 Weeks
Proportion of participants with pemphigus vulgaris and pemphigus foliaceus who had CRmin within 30 weeks, defined as the absence of new lesions and complete healing of established lesions while the participant was receiving prednisone at ≤10 mg/day for at least 8 weeks.
Time frame: up to 30 weeks treatment period
Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris Participants
Normalized Cumulative prednisone dose (NCPD, mg/kg/day) is the average daily intake of all weight-adjusted prednisone doses received during the study, taking into account the number of days in study
Time frame: Up to 30 weeks
Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris Participants
Time to complete remission (absence of new lesions and complete healing of established lesions) in participants with pemphigus vulgaris
Time frame: Up to 30 weeks
Time to Disease Control (DC) in Pemphigus Vulgaris (PV) Participants
Time to disease control in participants with pemphigus vulgaris (Absence of new lesions and the start of healing of established lesions)
Time frame: Up to 30 weeks
Normalized Cumulative Prednisone Dose During the Treatment Period in Pemphigus Vulgaris and Pemphigus Foliaceus Participants
Normalized Cumulative prednisone dose (NCPD, mg/kg/day) is the average daily intake of all weight-adjusted prednisone doses received during the study, taking into account the number of days in study
Time frame: Up to 30 weeks
Time to Complete Clinical Remission (CR) in Pemphigus Vulgaris and Pemphigus Foliaceus Participants
Time to complete remission (absence of new lesions and complete healing of established lesions) in participants with pemphigus vulgaris and pemphigus foliaceus
Time frame: Up to 30 weeks
Time to Disease Control in Pemphigus Vulgaris and Pemphigus Foliaceus Participants
Time to disease control in participants with pemphigus vulgaris and pemphigus foliaceus (Absence of new lesions and the start of healing of established lesions)
Time frame: Up to 30 weeks
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Investigator site 99 - US0010117
Miami, Florida, United States
Investigator site 78 - US0010109
Orlando, Florida, United States
Investigator site 127 - US0010155
West Lafayette, Indiana, United States
Investigator site 61 - US0010090
Minneapolis, Minnesota, United States
Investigator site 102 - US0010098
St Louis, Missouri, United States
...and 124 more locations