Ongoing Clinical Trials for Mast Cell Activation Syndrome
Currently, there are 2 ongoing clinical trials investigating new treatments for Mast Cell Activation Syndrome. These studies are being conducted across several European countries and are testing medications designed to help patients who have not responded well to standard treatments. The trials focus on reducing symptoms such as itching, flushing, fatigue, and depression that significantly impact daily life.
Clinical trial locations
- Belgium
- Bulgaria
- France
- Germany
- Italy
- Poland
- Spain
Study of TL-895 for Patients with Relapsed or Refractory Myelofibrosis or Indolent Systemic Mastocytosis
This trial is testing a medication called TL-895 in patients with Indolent Systemic Mastocytosis, a condition where mast cells accumulate abnormally in various tissues throughout the body. TL-895 is an oral medication taken as a film-coated tablet and works as a kinase inhibitor, targeting specific pathways involved in abnormal cell growth and survival.
Who can participate:
- Adults aged 18 years or older with a confirmed diagnosis of Indolent Systemic Mastocytosis based on bone marrow biopsy results following WHO diagnostic criteria
- Patients experiencing moderate-to-severe symptoms
- Those with adequate blood, liver, and kidney function
- Patients able to follow study procedures and attend regular monitoring visits
Who cannot participate:
- Patients with other serious health conditions that might interfere with the study
- Pregnant or breastfeeding women
- Those who have recently participated in another clinical trial
- Patients with a history of allergic reactions to similar medications
- Those with severe liver or kidney problems
- Patients with certain uncontrolled infections or unstable heart conditions
- Those who have had recent major surgery
Study focus: The main goal of this trial is to determine the best dose and treatment schedule for TL-895. Researchers will monitor participants regularly to assess symptom improvement and overall health changes. The study will track how well the medication reduces symptoms and will evaluate safety throughout the treatment period. This research is expected to conclude by the end of 2025 and aims to provide important information about whether TL-895 can be an effective treatment option for patients with this condition.
Study on Masitinib for Patients with Severe Mast Cell Activation Syndrome Unresponsive to Treatment
This trial is focused specifically on patients with severe Mast Cell Activation Syndrome who have not responded to standard treatments. The medication being tested is masitinib, a tyrosine kinase inhibitor taken orally as a coated tablet. Masitinib works by blocking certain enzymes that contribute to mast cell activation and proliferation.
Who can participate:
- Adults between 18 and 75 years old with severe symptoms affecting two or more body systems
- Patients with documented evidence of elevated serum tryptase during symptoms or abnormal levels of MCAS-related substances in blood or urine
- Those who have severe symptoms such as significant itching, frequent flushing, depression, or fatigue over a 14-day period
- Patients who have tried and failed at least two different treatments in the past two years, with each treatment lasting at least 8 weeks
- Those on a stable dose of Anti-H1 medication for at least 4 weeks before the study begins
- Patients weighing more than 45 kg with a Body Mass Index between 18 and 35
- Women of childbearing age and men with partners who can become pregnant must agree to use effective birth control during the study
Who cannot participate:
- Patients with conditions other than Mast Cell Activation Syndrome
- Those whose symptoms improve adequately with available treatments
- Pregnant or breastfeeding women
- Patients with other medical conditions or taking medications that might interfere with the study
- Those who have recently participated in another clinical trial
- Patients unwilling or unable to follow study procedures and attend regular visits
Study focus: This is a 24-week double-blind study, meaning neither participants nor researchers will know who receives the actual medication versus a placebo until the study concludes. The main goal is to evaluate whether masitinib can provide at least 50% improvement in key symptoms including itching, flushing, depression, and fatigue. Participants will have regular check-ups every four weeks to monitor treatment response and any side effects. This trial aims to determine whether masitinib can be a valuable treatment option for those with severe symptoms that have not responded to other therapies.
Summary
Both ongoing trials represent important research efforts to find effective treatments for patients with mast cell-related disorders who struggle with standard therapies. The first trial is part of a larger international study conducted across seven European countries, while the second trial is specifically focused in France.
Both studies are testing kinase inhibitors, which work by blocking specific molecular pathways involved in mast cell activation and growth. The TL-895 study is broader in scope, also including other blood disorders, and is expected to run through 2025. The masitinib study is more targeted, focusing exclusively on severe cases of Mast Cell Activation Syndrome with a 24-week treatment period.
These trials offer hope for patients who have not found relief with existing treatments, particularly those experiencing moderate-to-severe symptoms that significantly impact quality of life. Both studies require careful monitoring throughout the treatment period to ensure patient safety while evaluating effectiveness.
