NCT04598477

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

An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Pemphigus

• Status: Terminated
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 183 (actual)
• Sponsor: argenx · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This was a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK and PD of efgartigimod PH20 SC in adult PV or PF participants, who participated in antecedent trial ARGX-113-1904. This trial provided extension of efgartigimod PH20 SC treatment and retreatment options for participants who had been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to the placebo arm in trial ARGX-113-1904. The participants could also receive concomitant prednisone therapy. Investigators could increase or decrease the prednisone dose based on protocol-specified criteria. Trial ARGX-113-1905 evaluated the ability to (further) taper prednisone therapy and achieve Clinical Remission (CR) off therapy (CRoff), the ability to achieve CR and CR on minimal therapy (CRmin) for participants who had not yet achieved CR or CRmin, and the ability to treat flare; it also assessed patient outcome measures and the safety, PD, PK and immunogenicity of efgartigimod PH20 SC over the duration of trial. Study duration: Up to 60 weeks for participants who receive IMP administration up to 52 weeks and with a follow-up period of 8 weeks after the last IMP administration

Conditions

• Pemphigus Vulgaris
• Pemphigus Foliaceus

Interventions

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

Timeline

• Start date: 2021-07-15
• Primary completion: 2024-03-25
• Completion: 2024-03-25
• First posted: 2020-10-22
• Last updated: 2025-03-30
• Results posted: 2025-03-30

Locations

127 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