ADDRESS

• Research type

  Research Study

• Full title

  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) (ADDRESS)

• IRAS ID

  1003432

• Contact name

  Wim Parys

• Sponsor organisation

  argenx BV

• Eudract number

  2020-002915-23

• Clinicaltrials.gov Identifier

  NCT04598451

• Research summary

  Research Summary
  Pemphigus is a group of auto-immune diseases which cause painful blisters to develop on the skin and lining of the mouth, nose, throat and genitals. The blisters are fragile and can easily burst open, leaving areas of raw unhealed skin that are very painful and at risk of infections.

  Pemphigus vulgaris (PV) and pemphigus foliaceus (PF; also called superficial pemphigus) are 2 types of this disease. They are rare. Pemphigus can affect people of all ages, but most cases develop in older adults, both men and women, aged between 40-60 years. Pemphigus is not contagious and cannot be passed from one person to another.

  Pemphigus is chronic and difficult to control, with life-threatening potential. There is currently no cure for pemphigus. Oral corticosteroid treatment is often used initially to keep symptoms under control. However, high doses may be required and they have many side effects when taken at a high dose in the long term. Therefore, other medicines are often prescribed to allow the dose of steroids to be reduced.

  The aim of this study is to look at the effectiveness, safety and tolerability of the study drug (efgartigimod PH20 SC, which is being developed by argenx BV) in people with PV or PF. In clinical studies to date, efgartigimod has been well-tolerated in healthy adults and participants with pemphigus. It is hoped that the study drug may help improve the symptoms of pemphigus.

  This study is placebo-controlled and double-blind and about 150 people will take part. The study will last up to 41 weeks (approximately 10 months), including a screening period of up to 3 weeks and a treatment period of up to 30 weeks. After the treatment period, participants may have the choice to enter into an open-label extension study or enter into the 8 week follow-up period.

  Participants will undergo procedures such as blood samples, physical examination and electrocardiogram. Participants may also have the option to provide skin biopsies for a sub-study.

  Summary of Results
  A study to assess the safety and efficacy of a subcutaneous formulation of efgartigimod PH20 SC in adults with Pemphigus (Vulgaris or Foliaceus). The researchers compared the difference in the number of participants who had a symptom-free period between participants who received efgartigimod and those who received placebo. 44 of 124 participants who received efgartigimod had a symptom-free period within a 30-week period. 20 of 66 participants who received placebo had a symptom-free period within a 30-week period, A symptom-free period was defined as a period of 8 weeks during which the participants with pemphigus vulgaris had no new lesions and their existing lesions healed and were receiving a low dosage of prednisone (10 mg/day or less). The difference in the number of participants who had a symptom-free period between these two groups was not big enough to be considered 'statistically significant'.

• REC name

  North West - Greater Manchester Central Research Ethics Committee

• REC reference

  21/NW/0096

• Date of REC Opinion

  19 May 2021

• REC opinion

  Further Information Favourable Opinion