Ongoing Clinical Trials for Pemphigus
There are currently 2 ongoing clinical trials for pemphigus, a rare autoimmune skin disease that causes blisters and sores. These trials are testing new approaches to treatment, including low-dose immune therapy and personalized maintenance strategies. The trials are being conducted in Germany and France.
Clinical trial locations
- France
- Germany
Study on Low-Dose Aldesleukin for Patients with Pemphigus, Mucous Membrane Pemphigoid, Polymyositis, Dermatomyositis, and Primary Sclerosing Cholangitis
This trial is being conducted in Germany and is testing a treatment approach for several chronic inflammatory diseases, including pemphigus vulgaris and pemphigus foliaceus.
Who can participate: To be eligible, participants must be between 18 and 80 years old and have one of several conditions, including pemphigus vulgaris or pemphigus foliaceus. For those with pemphigus, the disease activity should be mild to moderate, with a score between 6 and 45 on a specific measurement scale called PDAI. Participants must meet specific disease activity criteria that show their condition is active but not too severe.
Who cannot participate: The trial excludes people who are pregnant or breastfeeding, have severe allergic reactions to similar treatments, have active infections requiring treatment, or have a history of cancer within the last five years (except certain skin cancers). People with significant heart problems, uncontrolled high blood pressure, severe liver or kidney disease, or recent participation in other investigational drug studies are also excluded.
What the trial is testing: The main focus of this study is to evaluate the safety and effectiveness of low-dose Interleukin-2 (also called IL-2 or aldesleukin) therapy. The treatment is given as an injection under the skin over an eight-week period. The goal is to understand how this medication affects the immune system and whether it leads to improvements in disease symptoms. The study is designed as a randomized, double-blind trial, which means participants are randomly assigned to different groups and neither they nor the researchers know who is receiving the actual treatment or a placebo.
The investigational drug: Interleukin-2 is being studied for its potential to help manage chronic inflammatory diseases by increasing the number of regulatory T cells in the body. These special immune cells help control the immune system and reduce inflammation. The medication is used in low doses to see if it can safely increase these beneficial cells and improve disease control.
Study on Personalized Maintenance Therapy with Rituximab and Corticosteroids for Patients with Pemphigus
This trial is being conducted in France and focuses specifically on pemphigus patients, testing a new personalized approach to long-term treatment maintenance.
Who can participate: Participants must be between 18 and 80 years old and enrolled in a social security or national health insurance plan. They must have a newly confirmed diagnosis of either pemphigus vulgaris or pemphigus foliaceus, based on specific skin or mucosal biopsy findings and laboratory tests. The disease must be moderate-to-severely active, with a PDAI score greater than 15. Participants must be able to receive standard treatment with corticosteroids and rituximab, and must be vaccinated against Covid-19 before joining the study. Women and men of childbearing potential must agree to use effective birth control methods during the treatment and for at least 12 months afterward.
Who cannot participate: The trial excludes people who do not have pemphigus vulgaris or pemphigus foliaceus. Those whose disease is not considered high-risk for relapse based on initial severity assessments, or whose disease activity cannot be properly monitored using specific blood tests for anti-desmoglein antibodies, cannot participate.
What the trial is testing: This study explores whether a personalized treatment plan can better prevent the return of symptoms over four years compared to current standard treatment. The personalized approach uses specific markers in the blood, called anti-desmoglein antibodies, to identify patients at higher risk of disease relapse. Those identified as high-risk may receive additional maintenance infusions of rituximab based on their individual risk level. The study will monitor participants over an extended period, with plans to continue until March 2032, to observe the effectiveness of the treatments in reducing relapse rates.
The investigational drugs: The trial uses rituximab, given as an intravenous infusion, along with corticosteroids such as prednisone, taken orally. Rituximab works by targeting specific immune system cells involved in the disease process, helping to reduce inflammation and prevent further damage to the skin and mucous membranes. Corticosteroids help reduce inflammation and suppress the immune system to control symptoms and prevent flare-ups. The innovative aspect of this trial is the personalized maintenance therapy approach, which uses antibody levels to determine when additional rituximab infusions are needed.
Summary
The two ongoing trials for pemphigus represent different approaches to managing this challenging autoimmune condition. The German trial takes a broader approach, including pemphigus patients alongside those with other inflammatory diseases, and tests a novel low-dose immune therapy using Interleukin-2. The French trial focuses exclusively on pemphigus patients and investigates a personalized maintenance strategy using established medications in a new way.
Both trials are designed for patients with active disease, though they target different severity levels. The German study accepts patients with mild to moderate disease activity, while the French study requires moderate-to-severely active disease. This reflects their different treatment goals: the German trial explores a new medication approach, while the French trial aims to optimize long-term maintenance therapy for more severely affected patients.
The geographic distribution shows research activity in Western Europe, with each country contributing one trial. The French trial is particularly notable for its long-term follow-up period extending to 2032, reflecting the chronic nature of pemphigus and the importance of preventing relapses over many years.
