ADDRESS+
Research type
Research Study
Full title
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 (ADDRESS+)
IRAS ID
1003460
Contact name
Wim Parys
Sponsor organisation
argenx BV
Eudract number
2020-002917-16
Clinicaltrials.gov Identifier
Research summary
Research Summary -
Pemphigus is a group of auto-immune diseases which cause painful blisters to develop on the skin & lining of the mouth, nose, throat & genitals. The blisters are fragile & can easily burst open, leaving areas of raw unhealed skin that are painful & at risk of infections.
Pemphigus vulgaris (PV) & 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 & women, aged between 40-60 years. Pemphigus is not contagious & cannot be passed from one person to another.
Pemphigus is chronic & difficult to control, with life-threatening potential. There is currently no cure for pemphigus. Oral corticosteroids are often used to initially keep symptoms under control. However high doses may be required & 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.
This is an open-label extension (OLE) study following on from the ARGX-113-1904 study. Participants who took part in the preceding placebo-controlled double-blind study may have the option to roll over into this OLE where all participants will receive the study drug (efgartigimod PH20 SC, developed by argenx BV). The aim of this study is to further look at the effectiveness, safety & tolerability of the study drug in people with PV or PF. In clinical studies to date, efgartigimod has been well-tolerated in healthy adults & participants with pemphigus. It is hoped that the study drug may help improve the symptoms of pemphigus.
The maximum number of participants in this study will be the number who entered into the preceding study (150 maximum). The study will last up to 60 weeks (approx 14 months), including a 52 week treatment period & 8 week treatment-free period.
Participants will undergo procedures such as blood samples, physical examination & electrocardiogram.
Lay Summary of Results -
"Researchers wanted to learn about new treatments for people with a skin disease called pemphigus. Pemphigus is a group of rare autoimmune skin conditions in which the body’s defense system attacks the connection between the skin and the moist lining (mucous membrane) of the digestive, respiratory, reproductive, and urinary tracts. This can cause painful lesions like sores, blisters, or bumps filled with fluid on the skin. These lesions can also be in mucous membranes, including the linings of the eyes, nose, mouth, throat, and genitals.
Researchers wanted to find out if efgartigimod would be a safe treatment to receive over a long time. Efgartigimod is a piece of a human antibody modified to bind to a protein called FcRn. Antibodies are proteins that fight and prevent infections. FcRn helps maintain antibody levels. Antibodies become harmful in some diseases, attacking the body. Antibody levels are reduced after efgartigimod binds to FcRn. This means that the levels of antibodies attacking the body are also reduced. A medicine called prednisone was also given to participants. Prednisone is a corticosteroid and the main treatment for pemphigus. The prednisone dosage could be changed based on pemphigus symptoms. People could join the study is they participated in an earlier study called ARGX-113-1904. Participants received egartigimod based on their pemphigus symptoms: those with symptoms received efgartigimod until their symptoms improved, If their symptoms worsened again, they received efgartigimod again; those without symptoms did not receive efgartigimod.
How many participants had serious adverse events? 20 of the 149 participants, of which 4 were possibly related to the study drug.
How many participants had adverse events including serious and of special interest? 107 of the 149 participants, of which 37 were possibly related to the study drug.
How many participants had adverse events of infection? 66 of the 149 participants.Researchers learned how efgartigimod could affect pemphigus symptoms. Researchers also collected safety data on efgartigimod. Efgartigimod was safe in participants with pemphigus. This is a summary of the main results from 1 study. Other studies may provide new information or different results. argenx stopped this follow-up safety study because the earlier study was unable to show that efgartigimod is an effective treatment option. No new studies are planned to learn about efgartigimod for the treatment of patients with pemphigus."
REC name
London - Hampstead Research Ethics Committee
REC reference
21/LO/0295
Date of REC Opinion
12 May 2021
REC opinion
Further Information Favourable Opinion