In short
Currently, there are 2 clinical trials investigating new treatment options for patients with mastocytosis. These studies are taking place in France, Spain, and Poland, focusing on patients with cutaneous and systemic forms of the condition who experience significant symptoms that have not responded well to standard treatments.
Clinical trial locations
- France
- Poland
- Spain
Study on Hydroxychloroquine for Patients with Cutaneous Mastocytosis or Indolent Systemic Mastocytosis with Skin Involvement
This trial is investigating whether hydroxychloroquine can help reduce troublesome symptoms in patients with two specific forms of the condition: isolated cutaneous mastocytosis, which affects only the skin, and indolent systemic mastocytosis with skin involvement. The study will run for 12 months and will compare the medication against a placebo to assess its effectiveness.
Main inclusion criteria: To participate, you must be over 18 years old and have either isolated cutaneous mastocytosis or indolent systemic mastocytosis with skin lesions. You need to experience moderate to severe symptoms, specifically itching with a score of 3 or higher on a scale of 0 to 10, or at least 5 episodes of flushing per week. You must also have a known skin KIT mutation, which is a specific genetic change related to the condition. Women and men of childbearing age must use effective contraception during treatment and for up to 8 months afterward.
Main exclusion criteria: You cannot participate if you have any other serious health condition that might interfere with the study, are pregnant or breastfeeding, have had an allergic reaction to hydroxychloroquine in the past, or are currently in another clinical trial. Other exclusions include a history of drug or alcohol abuse, severe liver or kidney disease, certain eye conditions, heart rhythm problems, or taking medications that might interact with hydroxychloroquine.
Focus of the trial: The study aims to evaluate how well hydroxychloroquine can reduce symptoms such as itching and flushing over 12 months. Participants will take the medication as a film-coated tablet daily, with the dosage adjusted to their body weight (between 6 to 6.5 mg per kilogram). Throughout the trial, researchers will monitor changes in symptoms and assess the safety of the treatment. The study will also track other symptoms related to mast cell activation, such as diarrhea and joint pain.
Investigational drug: Hydroxychloroquine is an antimalarial and immunomodulatory medication that is thought to work by reducing the activity of mast cells, potentially alleviating symptoms like itching and flushing.
Study on the Effectiveness and Safety of Masitinib for Patients with Severe Systemic Mastocytosis Unresponsive to Treatment
This trial is evaluating masitinib, a tyrosine kinase inhibitor, for patients with smouldering systemic mastocytosis or indolent systemic mastocytosis who have severe symptoms that do not respond to standard treatments. The study will last for 24 weeks, with a possible extension, and will compare masitinib with a placebo in a double-blind format.
Main inclusion criteria: You must have documented smouldering or indolent systemic mastocytosis with an excess of mast cells in at least two organs. You need to have severe symptoms over a 14-day period, such as intense itching, frequent flushing, or a high depression score. Importantly, you must have tried and not responded to at least two different treatments in the past two years, with each treatment lasting at least 8 weeks. You must be between 18 and 75 years old, weigh more than 45 kg, and have a Body Mass Index between 18 and 35. You also need to be on a stable dose of anti-H1 medication for at least 4 weeks before the study. Both you and your partner must use highly effective contraception during the study and for a specified period afterward.
Main exclusion criteria: You cannot participate if you have a different condition than mastocytosis or if you are not suffering from smouldering or indolent severe systemic forms with symptoms that do not respond to the best available treatment. Patients who are part of a vulnerable population requiring special protection or care are also excluded.
Focus of the trial: The primary goal is to compare the effectiveness of masitinib versus placebo in reducing symptoms and improving quality of life for patients with severe systemic mastocytosis. Participants will be randomly assigned to receive either masitinib (100 mg or 200 mg) or placebo as a coated tablet taken daily. The study includes regular visits to monitor treatment response and assess any side effects. The primary analysis will focus on response to treatment regarding three specific symptoms between weeks 8 and 24, while secondary analyses will evaluate biological and skin parameters as well as overall quality of life.
Investigational drug: Masitinib is an oral tyrosine kinase inhibitor that works by blocking certain enzymes involved in the growth and survival of mast cells. It is currently in phase III clinical trials for systemic mastocytosis.
Summary
The two ongoing clinical trials for mastocytosis offer different treatment approaches for patients struggling with this challenging condition. The first trial, conducted in France, focuses on hydroxychloroquine for patients with cutaneous or indolent systemic mastocytosis with skin involvement, running for 12 months. The second trial, taking place across France, Spain, and Poland, investigates masitinib for patients with more severe systemic forms who have not responded to other treatments, with a 24-week duration and possible extension.
Both studies target patients experiencing significant symptoms such as itching and flushing. Notably, the masitinib trial specifically requires participants to have previously failed at least two different treatments, indicating it is aimed at a more treatment-resistant patient population. The geographic distribution shows a concentration of research activity in France, which is participating in both trials, while Spain and Poland are involved in the masitinib study. These trials represent important efforts to find more effective treatments for patients whose symptoms remain inadequately controlled with current options.
