Ongoing Clinical Trials for Immune-Mediated Enterocolitis
This article provides information about ongoing clinical trials for immune-mediated enterocolitis, a condition where the immune system attacks the digestive tract causing inflammation. Currently, there is 1 clinical trial being conducted across multiple European countries, testing a new treatment called MAS825 for patients with specific genetic forms of this condition.
Clinical trial locations
- Czechia
- France
- Italy
- Spain
Study on MAS825 for Patients with Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations
This trial focuses on rare genetic conditions that cause the immune system to mistakenly attack the body, leading to repeated episodes of severe inflammation. These conditions include NLRC4-Gain of Function (also called autoinflammation with infantile enterocolitis or AIFEC), XIAP deficiency, and CDC42 mutations. These genetic disorders often cause symptoms such as recurrent fevers, abdominal pain, rashes, and inflammation in various parts of the body, particularly affecting the digestive tract.
Main inclusion criteria: Patients can join this study if they meet several requirements. They must weigh at least 3 kg (about 6.6 pounds) and can be male or female of any age, including children. Most importantly, participants must have a confirmed genetic diagnosis of one of the three conditions being studied: NLRC4-GOF, XIAP deficiency, or CDC42 mutation. For children, written consent must be provided by parents or legal guardians, and the child may also need to agree depending on their age and local regulations. Adults must provide their own written consent, or if unable, their legal representative can do so. Patients in Cohort 1 must show signs of active disease when starting treatment, with a medical history that matches the expected symptoms of these conditions. There is also a Cohort 2 for patients already receiving MAS825 through a special access program.
Main exclusion criteria: Patients cannot participate if they have other serious health conditions that could interfere with the study results or their safety. Those currently enrolled in another clinical trial are not eligible. Recent infections that might affect the study, pregnancy, or breastfeeding also exclude patients from participation. People with a history of allergic reactions to similar medications, those unable to follow study procedures or attend regular visits, and individuals with substance abuse issues are also excluded. Additionally, patients who have recently received certain treatments that could interfere with the study results cannot join.
Focus and goals: The main purpose of this trial is to test whether MAS825 can effectively prevent disease flares, which are periods when symptoms suddenly worsen. The study is divided into several periods. In the first period, all participants receive MAS825 through an injection into a vein. The medication works by blocking two specific proteins, IL-1 beta and IL-18, which play a key role in causing inflammation in these diseases. In the second period, called the randomized withdrawal phase, some patients will continue receiving MAS825 while others will receive a placebo (an inactive substance). Neither the patients nor their doctors will know which treatment they are receiving during this phase. This helps researchers determine whether MAS825 is truly effective. Throughout the study, doctors will closely monitor participants’ health, track inflammatory markers in their blood, measure disease activity using physician assessments, and record any improvements or side effects. The study will also look at whether patients can reduce their use of steroids, which are often used to control inflammation but can have significant side effects. The trial is expected to conclude by June 2027.
Investigational drug: The medication being tested is MAS825, a human IgG1 monoclonal antibody that targets both IL-1 beta and IL-18. These two proteins are involved in triggering and maintaining the excessive inflammation seen in these genetic conditions. By blocking these proteins, MAS825 aims to reduce inflammation and prevent the sudden worsening of symptoms known as flares. The medication is given as a solution through intravenous injection, with the dosage and frequency determined by the study protocol. This approach represents a new strategy for managing these rare autoinflammatory diseases, potentially offering patients better control of their symptoms and improved quality of life.
Summary
Currently, there is one clinical trial available for patients with immune-mediated enterocolitis related to specific genetic mutations. This trial is being conducted across four European countries: Czechia, France, Italy, and Spain, providing access to patients in multiple locations. The study focuses on testing MAS825, a novel monoclonal antibody treatment designed to block two key inflammatory proteins. This represents an important development for patients with NLRC4-GOF, XIAP deficiency, or CDC42 mutations, as these rare conditions currently have limited treatment options. The trial uses a rigorous scientific approach, including a placebo-controlled phase, to determine whether this new treatment can effectively prevent disease flares and improve patients’ quality of life. The multi-country design reflects the rarity of these conditions and the need for international collaboration to gather sufficient data. Patients interested in participating should discuss with their healthcare providers whether they meet the eligibility criteria and whether one of the participating countries is accessible to them.
