Masitinib for the Treatment of Severe Mast Cell Activation Syndrome
NCT ID: NCT05449444
Last Updated: 2023-02-06
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
PHASE2
72 participants
INTERVENTIONAL
2022-07-01
2024-12-31
Brief Summary
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Detailed Description
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Dosing scheme #1: Oral masitinib treatment at 3 mg/kg/day for 4 weeks, then a switch to 4.5 mg/kg/day for the remainder of the treatment period, versus placebo with a matching titration scheme. Randomization 2: 1 (Masitinib MCAS: Placebo MCAS).
Dosing scheme #2: Oral masitinib treatment at 3 mg/kg/day for 4 weeks, then a switch to 4.5 mg/kg/day for 4 weeks, then a second switch to 6 mg/kg/day for the remainder of the treatment period versus placebo treatment with a matching titration scheme. Randomization 2: 1 (Masitinib MCAS: Placebo MCAS)
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
DOUBLE
Study Groups
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Masitinib (4.5) & BSC
Masitinib 4.5 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) for 4 weeks, given orally twice daily, with a dose escalation to 4.5 mg/kg/day for the remainder of the treatment period. Each ascending dose titration is subjected to a safety control.
Masitinib 4.5 mg/kg/day
Masitinib 4.5 mg/kg/day
Best supportive care
Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, sodium cromoglicate, antidepressants, antileukotrienes, proton pump inhibitors, and corticosteroids.
Masitinib (6.0) & BSC
Masitinib 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) for 4 weeks, given orally twice daily, with a dose escalation to 4.5 mg/kg/day for4 weeks of treatment, then a second dose escalation to 6 mg/kg/day for the remainder of the treatment period. Each ascending dose titration is subjected to a safety control.
Best supportive care
Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, sodium cromoglicate, antidepressants, antileukotrienes, proton pump inhibitors, and corticosteroids.
Masitinib 6.0 mg/kg/day
Masitinib 6.0 mg/kg/day
Placebo & BSC
Placebo administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC). Participants receive a matched dose placebo, given orally twice daily.
Placebo
Matching placebo
Best supportive care
Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, sodium cromoglicate, antidepressants, antileukotrienes, proton pump inhibitors, and corticosteroids.
Interventions
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Masitinib 4.5 mg/kg/day
Masitinib 4.5 mg/kg/day
Placebo
Matching placebo
Best supportive care
Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, sodium cromoglicate, antidepressants, antileukotrienes, proton pump inhibitors, and corticosteroids.
Masitinib 6.0 mg/kg/day
Masitinib 6.0 mg/kg/day
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
* Patient with severe symptoms over the 14-day run-in period defined as at least one of the following: Pruritus score ≥ 9; Number of flushes per week ≥ 8; Hamilton rating scale for depression (HAMD-17) score ≥ 19
* Patient with documented treatment failures of his/her handicap(s) (within last two years) with at least two of the symptomatic treatments used at optimized dose.
* Patients must be on a stable dose of Anti-H1 for a minimum of 4 weeks before screening and should remain at a stable dose throughout the study period.
Exclusion Criteria
* Any change in the symptomatic treatment of MCAS, including systemic corticosteroids, or administration of any new treatment for MCAS within 4 weeks prior to screening.
* Patient with systemic indolent mastocytosis.
* Female patients who are pregnant or are breastfeeding.
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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Julien Rossignol, MD
Role: PRINCIPAL_INVESTIGATOR
Reference Centre for Mastocytosis (CEREMAST), Necker Hospital, Paris, France
Locations
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St Charles Clinical Research
Weldon Spring, Missouri, United States
Centre Hospitalier Universitaire Amiens-Picardie
Amiens, , France
Necker-Enfants Malades Hospital, Centre de référence des Mastocytoses (CEREMAST)
Paris, , France
CHU Toulouse
Toulouse, , France
Countries
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Central Contacts
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References
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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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2021-005406-96
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
AB20006
Identifier Type: -
Identifier Source: org_study_id
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