Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment
NCT ID: NCT04333108
Last Updated: 2023-05-06
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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UNKNOWN
PHASE3
140 participants
INTERVENTIONAL
2020-07-01
2025-06-30
Brief Summary
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Detailed Description
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Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Masitinib & BSC
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.
Masitinib
Masitinib 6 mg/kg/day
Best Supportive Care
Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, proton pump inhibitors (PPI), sodium cromoglycate, antidepressants, leukotriene antagonists and corticosteroids.
Placebo & BSC
Placebo Comparator:
Matching placebo administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC)
Placebo
Matching placebo
Best Supportive Care
Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, proton pump inhibitors (PPI), sodium cromoglycate, antidepressants, leukotriene antagonists and corticosteroids.
Interventions
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Masitinib
Masitinib 6 mg/kg/day
Placebo
Matching placebo
Best Supportive Care
Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, proton pump inhibitors (PPI), sodium cromoglycate, antidepressants, leukotriene antagonists and corticosteroids.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. An excess of mast cells or a presence of abnormal mast cells in at least two organs (among skin, bone-marrow and GI Tract).
3. Patient with documented systemic mastocytosis and evaluable disease based upon histological criteria
4. Patient with documented treatment failure of his/her symptom(s) (within the past 2 years) with at least two of the symptomatic treatments used at optimized dose: Anti H1, Anti H2, Proton pump inhibitor, Antidepressants, Cromoglycate Sodium, Antileukotriene.
5. Patient with severe symptoms of mastocytosis over the 14-day run-in period including at least one among pruritus, flushes, and depression: pruritus score ≥ 9, number of flushes per week ≥ 8, Hamilton rating scale for depression (HAMD-17) score ≥ 19.
Exclusion Criteria
2. Previous treatment with any Tyrosine Kinase Inhibitor
3. Treatment with any investigational agent within 8 weeks prior to screening.
18 Years
75 Years
ALL
No
Sponsors
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AB Science
INDUSTRY
Responsible Party
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Principal Investigators
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Cristina Bulai Livideanu, MD, MSc
Role: PRINCIPAL_INVESTIGATOR
Centre Hospitalier Universitaire, Service de Dermatologie, Toulouse -France
Locations
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Centre Hospitalier Universitaire d'Amiens
Amiens, , France
Hospital Jean-Minjoz
Besançon, , France
Grenoble University Hospital
Grenoble, , France
Hospital Claude Huriez
Lille, , France
Marseille University Hospital Timone
Marseille, , France
Centre de référence de Mastocytose (CEREMAST)
Paris, , France
Poitiers University Hospital
Poitiers, , France
Centre Hospitalier Universitaire
Toulouse, , France
University Hospital Charité
Berlin, , Germany
Erasmus University Medical Center
Rotterdam, , Netherlands
Medical University of Gdańsk
Gdansk, , Poland
The University Hospital in Krakow (Szpital Uniwersytecki w Krakowie)
Krakow, , Poland
University Hospital in Bucharest (Spitalul Universitar de Urgență București)
Bucharest, , Romania
Almazov National Medical Research Centre
Saint Petersburg, , Russia
Dnipropetrovsk Clinical Association of Emergency Medical Care of Dnipropetrovsk Regional
Dnipro, , Ukraine
Private Enterprise Private Manufacturing Company Acinus
Poltava, , Ukraine
Guy's and St Thomas' NHS Foundation Trust
London, , United Kingdom
Countries
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Central Contacts
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References
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Paul C, Sans B, Suarez F, Casassus P, Barete S, Lanternier F, Grandpeix-Guyodo C, Dubreuil P, Palmerini F, Mansfield CD, Gineste P, Moussy A, Hermine O, Lortholary O. Masitinib for the treatment of systemic and cutaneous mastocytosis with handicap: a phase 2a study. Am J Hematol. 2010 Dec;85(12):921-5. doi: 10.1002/ajh.21894.
Lortholary O, Chandesris MO, Bulai Livideanu C, Paul C, Guillet G, Jassem E, Niedoszytko M, Barete S, Verstovsek S, Grattan C, Damaj G, Canioni D, Fraitag S, Lhermitte L, Georgin Lavialle S, Frenzel L, Afrin LB, Hanssens K, Agopian J, Gaillard R, Kinet JP, Auclair C, Mansfield C, Moussy A, Dubreuil P, Hermine O. Masitinib for treatment of severely symptomatic indolent systemic mastocytosis: a randomised, placebo-controlled, phase 3 study. Lancet. 2017 Feb 11;389(10069):612-620. doi: 10.1016/S0140-6736(16)31403-9. Epub 2017 Jan 7.
Latham BD, Oskin DS, Crouch RD, Vergne MJ, Jackson KD. Cytochromes P450 2C8 and 3A Catalyze the Metabolic Activation of the Tyrosine Kinase Inhibitor Masitinib. Chem Res Toxicol. 2022 Sep 19;35(9):1467-1481. doi: 10.1021/acs.chemrestox.2c00057. Epub 2022 Sep 1.
Other Identifiers
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2016-001447-39
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AB15003
Identifier Type: -
Identifier Source: org_study_id
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