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RekrutierungTherapeutische Bestätigung (Phase III)Interventionell

A 24-week with possible extension, prospective, multicenter, randomized, double blind, placebo-controlled, 2-parallel group with a randomization 1:1, phase III study to compare efficacy and safety of oral masitinib to placebo in treatment of patients with Smouldering or Indolent Severe Systemic mastocytosis with handicap, unresponsive to optimal symptomatic treatment

Systemic mastocytosis is a rare condition where the body produces too many mast cells, which are a type of immune cell. When these cells build up in organs and skin, they can cause severe, disabling symptoms such as extreme tiredness, skin flushing, bone pain, and gut problems that standard medicines do not always ease. This Phase 3 clinical trial is testing whether an oral medicine called masitinib can help adults whose severe symptoms persist despite current treatments. Participants take tablets daily for 24 weeks. Half will receive masitinib and half will receive a dummy pill (placebo) to ensure a fair test. Doctors will monitor changes in severe symptoms, skin issues, blood test results, and overall wellbeing. If the trial goes well, participants may have the opportunity to continue treatment in a longer extension study.

Auf einen Blick

Status
Rekrutierung
Phase
Therapeutische Bestätigung (Phase III)
Sponsor
Ab Science
Einschreibeziel
70
Starten
30 Jan 2025

Worum geht es in dieser Studie?

Mast cells are an important part of our immune system, usually helping to fight infections and control allergic reactions. In systemic mastocytosis, too many mast cells gather in different tissues and organs. These cells can become overactive and release chemicals that trigger uncomfortable and disruptive symptoms throughout the body, including severe exhaustion, hot flushes, stomach pain, and itchy skin patches.

For many people, standard treatments such as antihistamines are not enough to keep these symptoms under control. When symptoms continue to get in the way of working, socialising, or simple daily tasks, doctors describe this as having a disabling impact or handicap. Finding targeted medicines that can calm down these overactive mast cells is an important goal for researchers.

This trial is looking closely at masitinib, a targeted medicine taken by mouth. Doctors want to see if masitinib can safely reduce the burden of these troublesome symptoms over 24 weeks. Because it is a Phase 3 trial, it is one of the final stages of testing to confirm whether the medicine works well enough and is safe enough to be made widely available in the future.

Wichtige Erkenntnisse

  • Tests a daily tablet called masitinib for severe systemic mastocytosis.
  • Aimed at adults whose symptoms remain disabling despite standard treatments.
  • Runs for an initial 24 weeks, with a possible extension phase.
  • Half of the participants receive masitinib and half receive a dummy pill (placebo).
  • Participation is entirely voluntary and you can stop at any point.

Wer könnte berechtigt sein?

This trial is for adults aged 18 and older who have been diagnosed with indolent or smouldering systemic mastocytosis. These are forms of the condition that develop slowly but can still cause very troublesome daily symptoms.

To be considered, participants must have severe, disabling symptoms—such as intense fatigue, frequent flushing, severe itching, or persistent digestive problems—that have not improved enough with standard symptom-relieving medications.

The trial team will check each person's general health to make sure taking part is safe. People who have certain other serious health conditions, significant liver or kidney issues, or who are pregnant or breastfeeding may not be able to join.

Könnte diese Studie für Sie geeignet sein?

Beantworten Sie diese kurzen Fragen, um zu sehen, ob Sie möglicherweise berechtigt sind. Dies ist nur ein Leitfaden – das Forschungsteam trifft die endgültige Entscheidung.

  1. Are you aged 18 or older?
  2. Do you have a confirmed diagnosis of indolent or smouldering systemic mastocytosis?
  3. Do your symptoms significantly disrupt your normal daily activities?
  4. Have standard treatments failed to control your symptoms?
  5. Are you able to take tablets and attend regular hospital check-ups over 24 weeks?
Beantworten Sie jede Frage, um Ihr Ergebnis zu sehen.

Was beinhaltet die Teilnahme?

Taking part lasts for at least 24 weeks, with the possibility of staying on the study longer if it helps. Participants take their assigned study tablets once or twice a day by mouth. Half of the people taking part will receive masitinib and half will receive an inactive dummy pill (placebo). Neither you nor your study doctor will know which treatment you are taking, which helps keep the results unbiased.

You will need to attend regular clinic visits at the hospital. During these visits, the research team will carry out standard health checks, including blood tests, physical examinations, and skin evaluations. You will also be asked to complete questionnaires about your symptoms, everyday activities, and overall quality of life. Travel expenses are often supported by the study team.

Potenzielle Risiken und Vorteile

You may experience an improvement in your symptoms if you receive masitinib, and the information collected will help doctors learn more about treating this rare condition in the future. However, there is no guarantee that your symptoms will improve, and you might receive the placebo tablet. Masitinib can also cause side effects, which may include nausea, diarrhoea, swelling, muscle cramps, or skin rashes. Your health will be monitored very closely throughout the trial, and you can choose to leave the study at any time without your regular NHS medical care being affected.

Standorte (3)

Einige Standortangaben sind ungefähr. Wir arbeiten daran, dies zu verbessern — bitte überprüfen Sie die Angaben mit dem Versuchsteam, bevor Sie reisen.
  • —
    Unbestätigt
    Polen
  • —
    Unbestätigt
    Frankreich
  • —
    Unbestätigt
    Spanien

Häufige Fragen

What is masitinib?

Masitinib is an oral medicine designed to block specific signals inside mast cells, aiming to calm them down and stop them from causing severe symptoms.

Was ist ein Placebo?

A placebo is a dummy tablet that looks identical to the real medicine but contains no active ingredients. It helps researchers see whether changes in health are truly caused by the study drug.

Can I choose whether I receive masitinib or the placebo?

No. A computer assigns participants to masitinib or placebo by chance (like flipping a coin) so the trial remains completely fair and scientifically accurate.

Will I have to stop taking my usual medicines?

The study doctor will review your regular medicines with you to check which ones are safe to continue alongside the study treatment.

What happens after the 24 weeks are finished?

Depending on your response and how the trial is set up, there may be an extension period where eligible participants can continue taking the study medication.

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