A study to assess the efficacy and safety of efgartigimod PH20 SC in adults with systemic sclerosis

• Research type

  Research Study

• Full title

  A Randomized, Double-Blinded, Placebo-Controlled, Phase 2, Parallel-Group Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacodynamics, Pharmacokinetics, and Immunogenicity of Efgartigimod PH20 SC in Adult Participants With Systemic Sclerosis

• IRAS ID

  1011034

• Contact name

  Sabine Coppieters

• Contact email

  regulatory@argenx.com

• Sponsor organisation

  argenx BV

• Clinicaltrials.gov Identifier

  NCT06655155

• Research summary

  Systemic sclerosis (SSc) is a rare, chronic, autoimmune, connective tissue disease with one of the highest mortality rates among rheumatic diseases. It is characterized by inflammation, progressive fibrosis of the skin and internal organs, and severe vasculopathy. The disease causes moderate-to-severe skin problems, and day-to-day difficulties in daily activities.
  The main purpose of this study is to learn more about how the study drug called efgartigimod works and whether it is efficacious and safe for people with SSc. To understand how well Efgartigimod works, it will be compared with a placebo.
  Approximately 81 participants will be in the study at different study sites worldwide.
  Efgartigimod helps remove autoantibodies (these are types of proteins produced by our immune system that attack the patient’s own body). The study aims to understand what will happen to the body when Efgartigimod lowers the levels of autoantibodies to balance the immune system and may reduce the symptoms of SSc. The study drug (Efgartigimod or placebo) is given as an injection under the skin using a needle attached to a prefilled syringe. The patient or a caregiver may give the injections at the site or at home if the study doctor agrees.
  Participants will be assigned to one of two groups and receive either Efgartigimod PH20 SC or placebo by chance (this is called “randomization”). But neither participants nor study doctors will know which study medicine participants receive (this is called “double-blind”).
  Some participants will be interviewed (“exit interview”) within 4 weeks after the last dose of the study about their experience of the study.
  At most visits, patients give blood and urine samples, have physical examinations, electrocardiograms, and lung function tests, and answer questionnaires. A skin biopsy is optional.
  The study will last about 59 weeks

• REC name

  London - Hampstead Research Ethics Committee

• REC reference

  25/LO/0033

• Date of REC Opinion

  18 Feb 2025

• REC opinion

  Further Information Favourable Opinion