Ongoing Clinical Trials for Immune System Disorders
There are currently 5 clinical trials investigating new treatments and therapeutic approaches for immune system disorders. These studies are being conducted across several European countries, including Italy, France, Sweden, Netherlands, Spain, and Czechia. The trials focus on conditions such as systemic sclerosis, ulcerative colitis, sarcoidosis, and rare autoinflammatory diseases caused by genetic mutations.
Clinical trial locations
- Czechia
- France
- Italy
- Study on Early Diffuse Cutaneous Systemic Sclerosis: Comparing Stem Cell Transplantation with Cyclophosphamide, Filgrastim, and Mycophenolate Mofetil
- Study on MAS825 for Patients with Monogenic IL-18 Driven Autoinflammatory Diseases, Including NLRC4-GOF, XIAP Deficiency, or CDC42 Mutations
- Study on Predictive Factors for Response to Tofacitinib in Patients with Active Ulcerative Colitis
- Study of Hydroxychloroquine Safety and Effectiveness in Patients with Early Systemic Sclerosis
- Netherlands
- Spain
- Sweden
Study of Hydroxychloroquine Safety and Effectiveness in Patients with Early Systemic Sclerosis
This trial is investigating whether adding hydroxychloroquine to standard treatments can help patients with early-stage systemic sclerosis. Systemic sclerosis is a rare autoimmune condition that causes hardening of the skin and can affect internal organs.
Main inclusion criteria: To participate, you must be at least 18 years old with a confirmed diagnosis of systemic sclerosis. The disease must have started within the last 5 years, not counting Raynaud’s phenomenon. You need to have been on stable treatment for at least 4 weeks before joining. You must either have never taken hydroxychloroquine before or stopped it at least 16 weeks prior to the study.
Main exclusion criteria: You cannot participate if you are under 18 or over 65 years old, have a known allergy to hydroxychloroquine, are pregnant or breastfeeding, or have significant heart, kidney, or liver problems. Other reasons for exclusion include retinal eye problems, uncontrolled diabetes, active infections, history of seizures, or severe muscle weakness conditions.
Focus of the trial: The study will compare patients receiving hydroxychloroquine tablets with those receiving a placebo over 52 weeks. Both groups will continue their standard treatments. Doctors will monitor skin changes, blood vessel function, pain levels, morning stiffness, and fatigue throughout the year-long study period.
Investigational drug: Hydroxychloroquine is taken orally in tablet form. The daily dose is calculated based on body weight, up to a maximum of 400 mg per day. This medication is commonly used to treat malaria and certain autoimmune conditions, and it works by reducing inflammation and modifying immune system function.
Study on Early Diffuse Cutaneous Systemic Sclerosis: Comparing Stem Cell Transplantation with Cyclophosphamide, Filgrastim, and Mycophenolate Mofetil
This trial compares two treatment approaches for diffuse cutaneous systemic sclerosis: autologous hematopoietic stem cell transplantation versus immunosuppressive medications. The condition causes hardening and tightening of the skin and can affect internal organs.
Main inclusion criteria: You must be between 18 and 65 years old with a confirmed diagnosis of diffuse cutaneous systemic sclerosis. The disease must have started within the last 3 years, not counting Raynaud’s phenomenon. You need either a skin score of 15 or higher indicating diffuse skin involvement, or significant organ involvement affecting the lungs, kidneys, or heart. Alternatively, if the disease started within 1 year, you need a skin score greater than 10 with high risk of organ involvement.
Main exclusion criteria: You cannot participate if your condition doesn’t meet the specific diagnostic criteria, you’re outside the age range, unable to follow study procedures, have other serious health conditions that would interfere with the study, are pregnant or breastfeeding, or have recently participated in another clinical trial.
Focus of the trial: Participants will be randomly assigned to receive either stem cell transplantation or immunosuppressive medications. The study will monitor survival rates and prevention of major organ failure. Regular assessments will track various health indicators throughout the study period.
Investigational treatments: The stem cell transplantation group will receive preparatory medications including cyclophosphamide by injection, filgrastim through subcutaneous administration, mycophenolate mofetil taken orally, and anti-T lymphocyte immunoglobulin through intravenous infusion. The comparison group will receive immunosuppressive medications designed to suppress the immune system.
Study on Predictive Factors for Response to Tofacitinib in Patients with Active Ulcerative Colitis
This trial aims to identify factors that predict how patients with active ulcerative colitis will respond to treatment with tofacitinib. Ulcerative colitis causes inflammation and sores in the lining of the large intestine and rectum.
Main inclusion criteria: You must be over 18 years old with a confirmed diagnosis of ulcerative colitis. Your disease must be moderately to severely active, measured by a Mayo score of 6-12, including a rectal bleeding score between 1 and 3. You need an endoscopic Mayo subscore of 2-3. You must have experienced failure or intolerance to at least one standard treatment, such as glucocorticoids, azathioprine, 6-mercaptopurine, or biologic drugs. Women of childbearing age must use effective contraception throughout the study and for 15 weeks after stopping the medication.
Main exclusion criteria: You cannot participate if you don’t have ulcerative colitis, are outside the specified age range, cannot follow study procedures or provide informed consent, have other significant health conditions that might interfere with the study, are pregnant or breastfeeding, have recently participated in another trial, have a known allergy to tofacitinib, are taking medications that interfere with the study drug, or have a history of certain infections.
Focus of the trial: The study will analyze immune system responses through tissue biopsies to understand which patients are more likely to benefit from tofacitinib therapy. This research could help doctors determine the best treatment options for individuals with this chronic inflammatory condition.
Investigational drug: Tofacitinib is taken orally as a 10 mg film-coated tablet. It works by blocking certain enzymes called Janus kinases, which play a role in the inflammatory process. By inhibiting these enzymes, tofacitinib helps reduce inflammation and alleviate symptoms associated with ulcerative colitis.
Study on Stopping Infliximab, Methotrexate, and Azathioprine in Patients with Sarcoidosis in Remission
This trial evaluates the effects of stopping infliximab treatment in patients with sarcoidosis who have achieved remission. Sarcoidosis is a disorder affecting the immune system that causes inflammation in various organs.
Main inclusion criteria: You must be at least 18 years old and affiliated with the National French social security system. You need a clinical and radiological presentation consistent with sarcoidosis, with non-caseating granulomas in at least one organ. Other causes of granulomas must be ruled out. You must have been on infliximab treatment for at least 6 months, with a steroid dosage of 10 mg per day or less for at least 6 months. Your disease must show no activity for at least 6 months, indicated by an ePOST score of 0, with normal ACE and serum calcium levels.
Main exclusion criteria: Patients with other immune system disorders that would interfere with the study cannot participate.
Focus of the trial: The study will compare two strategies for maintaining remission. Some participants will continue with their current treatment, while others will stop taking infliximab. Researchers will monitor how many participants experience a major relapse over a 12-month period to understand the best way to manage the condition after initial treatment.
Investigational drugs: Infliximab is administered through intravenous infusion. It’s a TNF-alpha antagonist that works by blocking a protein called TNF-alpha, which causes inflammation in the body. The study also involves methotrexate and azathioprine, which are taken in tablet form to help suppress the immune system.
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 autoinflammatory diseases caused by specific genetic mutations, including NLRC4-Gain of Function, XIAP deficiency, and CDC42 mutations. These conditions cause the immune system to mistakenly attack the body, leading to excessive inflammation.
Main inclusion criteria: Patients must weigh at least 3 kg. For children, written agreement from parents or legal guardians is required, and the child may also need to agree depending on local rules. Adults must provide written consent themselves or through a legal representative. For Cohort 1, you must have a genetic diagnosis of NLRC4-GOF, XIAP deficiency, or CDC42 mutation, with a history and medical tests matching these conditions. At the start of treatment, there must be signs of active disease. For Cohort 2, you must have a genetic diagnosis and be receiving treatment with MAS825 in a special program managed by Novartis.
Main exclusion criteria: You cannot participate if you have other serious health conditions that could interfere with the study, are currently in another clinical trial, have had a recent infection affecting study results, are pregnant or breastfeeding, have a history of allergic reactions to similar medications, cannot follow study procedures or attend visits, have a history of substance abuse, or have received certain recent treatments that could affect the study.
Focus of the trial: The study will evaluate how effective and safe MAS825 is in preventing disease flares. Some participants will receive MAS825, while others will receive a placebo. The study is conducted over several periods, closely monitoring participants’ health and response to treatment.
Investigational drug: MAS825 is a human IgG1 monoclonal antibody that targets IL-1 beta and IL-18, proteins involved in the inflammatory process. It is administered as an injection into a vein. By blocking these proteins, MAS825 aims to reduce inflammation and prevent disease flares in patients with these rare autoinflammatory conditions.
Summary
The five ongoing clinical trials for immune system disorders reflect a diverse range of research approaches across multiple European countries. Italy hosts the most trials with four studies, followed by France with two, while Czechia, Spain, Sweden, and the Netherlands each host one trial.
The research covers several distinct conditions within the immune system disorder spectrum. Two trials focus on systemic sclerosis, investigating different treatment approaches: hydroxychloroquine as an add-on therapy for early-stage disease, and comparing stem cell transplantation with immunosuppressive medications for diffuse cutaneous forms. Another trial examines ulcerative colitis treatment with tofacitinib, while a fourth investigates maintenance therapy strategies for sarcoidosis patients in remission. The fifth trial addresses rare genetic autoinflammatory diseases caused by specific mutations.
Treatment approaches vary considerably, ranging from traditional immunosuppressive medications to innovative therapies like stem cell transplantation and monoclonal antibodies. The trials employ different study designs, including randomized controlled trials, comparison studies, and observational research, all aimed at improving understanding and management of these complex immune system disorders.