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Tyrosine Kinase Inhibitor

Masitinib for Systemic Mastocytosis

Phase 3

Recruiting

Led By Cristina Bulai Livideanu, MD, MSc

Research Sponsored by AB Science

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patient with documented systemic mastocytosis and evaluable disease based upon histological criteria

An excess of mast cells or a presence of abnormal mast cells in at least two organs (among skin, bone-marrow and GI Tract)

Timeline

Screening 3 weeks

Treatment Varies

Follow Up 24 weeks

Awards & highlights

Study Summary

This trial will study whether the drug masitinib can help treat patients with smouldering or indolent systemic mastocytosis who have severe symptoms that haven't been helped by other treatments.

Who is the study for?

This trial is for patients with severe symptoms of systemic mastocytosis, specifically the smoldering or indolent types, who haven't responded to treatments like antihistamines or antidepressants. They must have a certain level of itching, flushing, or depression and evidence of abnormal mast cells in at least two organs.Check my eligibility

What is being tested?

The study compares masitinib—a drug that targets specific enzymes—to a placebo in treating systemic mastocytosis when usual symptom relief doesn't work. Participants will receive either masitinib or a placebo alongside their supportive care regimen.See study design

What are the potential side effects?

Masitinib may cause side effects such as nausea, rash, fatigue, digestive issues and muscle pain. Since it's an enzyme inhibitor, there might also be risks related to blood cell counts and liver function.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have been diagnosed with systemic mastocytosis based on tissue analysis.

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I have too many or abnormal mast cells in at least two organs.

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I have severe itching, flushing, or depression due to mastocytosis.

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I have a type of mastocytosis called either Smouldering or Indolent.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ 24 weeks

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~24 weeks

This trial's timeline: 3 weeks for screening, Varies for treatment, and 24 weeks for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.

Primary outcome measures

Cumulative response (3R75%)

Secondary outcome measures

Cumulative response

Cumulative response (2R75%)

Cumulative response (4R75%)

Trial Design

2Treatment groups

Experimental Treatment

Placebo Group

Group I: Masitinib & BSCExperimental Treatment2 Interventions

Experimental Arm: Masitinib (titration to 6.0 mg/kg/day) administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC). Participants receive masitinib (3.0 mg/kg/day), given orally twice daily, with a dose escalation to 4.5 mg/kg/day after 4 weeks of treatment, followed by dose escalation to 6.0 mg/kg/day after 4 weeks of treatment. Each ascending dose titration is subjected to a safety control.

Group II: Placebo & BSCPlacebo Group2 Interventions

Placebo Comparator: Matching placebo administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC)

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Masitinib

2011

Completed Phase 3

~4480

0 / 4Eligibility criteria met

Find a Location

Who is running the clinical trial?

AB ScienceLead Sponsor

38 Previous Clinical Trials

15,582 Total Patients Enrolled

Cristina Bulai Livideanu, MD, MScPrincipal InvestigatorCentre Hospitalier Universitaire, Service de Dermatologie, Toulouse -France

Media Library

Masitinib (Tyrosine Kinase Inhibitor) Clinical Trial Eligibility Overview. Trial Name: NCT04333108 — Phase 3

Systemic Mastocytosis Research Study Groups: Masitinib & BSC, Placebo & BSC

Systemic Mastocytosis Clinical Trial 2023: Masitinib Highlights & Side Effects. Trial Name: NCT04333108 — Phase 3

Masitinib (Tyrosine Kinase Inhibitor) 2023 Treatment Timeline for Medical Study. Trial Name: NCT04333108 — Phase 3

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.

Is Masitinib known to have negative effects on human health?

"Masitinib's safety is well-documented, as this is a Phase 3 trial. This means that, in addition to efficacy data, there are multiple rounds of safety data supporting Masitinib."

Answered by AI

Is age a factor in this clinical trial's recruitment process?

"From the language in the inclusion criteria, it appears that this trial is only for people aged 18-75. There are 2 studies separate from this one which involve patients who are either under 18 or over 65."

Answered by AI

Is this a unique medical study?

"Masitinib's research journey started in 2020 with a study of 200 people. AB Science was the first to sponsor this clinical trial. Since then, Masitinib has gone through Phase 2 drug approval and there are now 4 active studies being conducted in 12 different cities across 14 countries."

Answered by AI

Do we have any previous data to suggest that Masitinib is effective?

"There are 4 ongoing studies for Masitinib, 2 of which have reached the third and final phase. The primary locations conducting these trials are in Paris and Minnesota; however, there are a total of 65 sites running these clinical trials."

Answered by AI

Recent research and studies

American Journal of HematologyJournal

Masitinib for the Treatment of Systemic and Cutaneous Mastocytosis with Handicap: A Phase 2a Study

Paul, Carle, Beatrix Sans, Felipe Suarez, Philippe Casassus, Stéphane Barete, Fanny Lanternier, Catherine Grandpeix-Guyodo, et al.. 2010. “Masitinib for the Treatment of Systemic and Cutaneous Mastocytosis with Handicap: A Phase 2a Study”. American Journal of Hematology. Wiley. doi:10.1002/ajh.21894.

The LancetJournal

Masitinib for Treatment of Severely Symptomatic Indolent Systemic Mastocytosis: A Randomised, Placebo-controlled, Phase 3 Study

Lortholary, Olivier, Marie Olivia Chandesris, Cristina Bulai Livideanu, Carle Paul, Gérard Guillet, Ewa Jassem, Marek Niedoszytko, et al.. 2017. “Masitinib for Treatment of Severely Symptomatic Indolent Systemic Mastocytosis: A Randomised, Placebo-controlled, Phase 3 Study”. The Lancet. Elsevier BV. doi:10.1016/s0140-6736(16)31403-9.

American Journal of HematologyJournal

Masitinib for the Treatment of Systemic and Cutaneous Mastocytosis with Handicap: A Phase 2a Study

The LancetJournal

Masitinib for Treatment of Severely Symptomatic Indolent Systemic Mastocytosis: A Randomised, Placebo-controlled, Phase 3 Study