NCT04598451

A Study to Assess the Efficacy and Safety of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)

A Randomized, Double-Blinded, Placebo-Controlled Trial to Investigate the Efficacy, Safety, and Tolerability of Efgartigimod PH20 SC in Adult Patients With Pemphigus (Vulgaris or Foliaceus)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 222 (actual)
• Sponsor: argenx · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

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

Conditions

• Pemphigus Vulgaris
• Pemphigus Foliaceus

Interventions

Type	Name	Description
BIOLOGICAL	efgartigimod PH20 SC	Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer
OTHER	Placebo	Subcutaneous injection of placebo
DRUG	prednisone	Oral prednisone tablets

Timeline

• Start date: 2020-12-01
• Primary completion: 2023-08-22
• Completion: 2023-08-22
• First posted: 2020-10-22
• Last updated: 2024-10-01
• Results posted: 2024-10-01

Locations

134 sites across 21 countries: United States, Australia, Bulgaria, China, France, Georgia, Germany, Greece, Hungary, India, Israel, Italy, Japan, Poland, Romania, Russia, Serbia, Spain, Turkey (Türkiye), Ukraine, United Kingdom

Regulatory

• FDA-regulated drug study