A phase 2/3 study of efgartigimod PH20 SC in adult participants with bullous pemphigoid
A Phase 2/3, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Group Study to Investigate the Efficacy and Safety of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid
Bullous pemphigoid (BP) is an autoimmune skin condition, it starts with an itchy raised rash. As the condition develops, large blisters can form on the skin. The blisters can grow big and may contain blood, if the blister burst open, they leave areas of raw unhealed skin that are very painful and at risk of infection.
BP can affect people of all ages, most cases develop in older adults, both men and women, aged 65 years and over.
BP is chronic and difficult to control, with life-threatening potential and can seriously affect quality of life. Currently there are no licenced treatments specifically for BP. The standard of care treatment is oral corticosteroid treatment is often used to keep symptoms under control. Unfortunately corticosteroid treatment in BP patients typically causes the onset of additional conditions which can be severe or life threatening especially in the elderly. Other medicines are often prescribed to allow the dose of corticosteroids 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 BP. It is hoped that the study drug may help improve the symptoms of BP.
This study is placebo-controlled and double-blind and consists of 2 parts (Part A: phase 2 proof-of-concept and Part B: phase 3 confirmatory). About 160 people will take part. Each part of the study will last up to 46 weeks (10-11 months), including a screening period of up to 3 weeks and a treatment period of up to 36 weeks. After the treatment period, participants may have the choice to enter into an extension study or end the follow-up period.
Participants will undergo procedures such as blood samples, physical examination and electrocardiogram.
At selected study sites, participants may also have the option to participate in a vaccination response substudy, the influence of the study drug on vaccines received during the study will be investigated.
South Central - Hampshire B Research Ethics Committee
Date of REC Opinion
10 Jun 2022
Further Information Favourable Opinion