Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis
NCT ID: NCT00814073
Last Updated: 2019-12-03
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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COMPLETED
PHASE3
135 participants
INTERVENTIONAL
2008-12-31
2015-11-30
Brief Summary
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Detailed Description
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A study protocol amendment restricted enrolment to patients with severe indolent and smoldering systemic mastocytosis. The objective of this phase 3 study was therefore to evaluate masitinib efficacy and safety in severe systemic mastocytosis patients, with or without D816V mutation of c-Kit. The primary objective of the phase 3 study was to detect a statistically significant difference between masitinib (plus optimal concomitant symptomatic treatments) and placebo (plus optimal concomitant symptomatic treatments) in cumulative response on four severe symptoms, referred to also as handicaps.
Conditions
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
QUADRUPLE
Study Groups
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Masitinib & BSC
Masitinib (6 mg/kg/day) administered as an add-on to optimal concomitant symptomatic treatment (i.e. best supportive care, BSC)
Masitinib
Masitinib 6 mg/kg/day
Best Supportive Care
Optimal concomitant symptomatic treatments. Includes: H1- and H2-antihistamines, proton pump inhibitors (PPI), sodium cromoglicate, antidepressants, leukotriene antagonists, interferon-alpha, 2-CdA, and corticosteroids.
Placebo & BSC
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 cromoglicate, antidepressants, leukotriene antagonists, interferon-alpha, 2-CdA, 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 cromoglicate, antidepressants, leukotriene antagonists, interferon-alpha, 2-CdA, and corticosteroids.
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
2. Patient with documented mastocytosis and evaluable disease based upon histological criteria: typical infiltrates of mast cells in a multifocal or diffuse pattern in skin and/or bone marrow biopsy
3. Patient with documented treatment failure of his/her handicap(s) with at least one of the following therapy used at optimized dose: Anti H1, Anti H2, Proton pump inhibitor, Osteoclast inhibitor, Cromoglycate Sodium, Antileukotriene
4. Handicapped status defined as at least two of the following handicaps, including at least one among pruritus, flushes, depression and fatigue: pruritus score ≥ 9, number of flushes per week ≥ 8, Hamilton rating scale for depression (HAMD-17) score ≥ 19, number of stools per day ≥ 4, number of mictions per day ≥ 8, Fatigue Impact Scale total score (asthenia) ≥ 75
5. Patients with OPA ≥ 2 (moderate to intolerable general handicap)
6. ECOG ≤ 2
7. Patient with adequate organ function
Exclusion Criteria
2. Previous treatment with any Tyrosine Kinase Inhibitor
3. Patient with recent cardiac history of: Acute coronary syndrome, Acute heart failure, Significant ventricular arrhythmia; patient with cardiac failure class III or IV; Syncope without known aetiology within 3 months, uncontrolled severe hypertension.
4. Patient with any condition that the physician judges could be detrimental to subjects participating in this study; including any clinically important deviations from normal clinical laboratory values or concurrent medical events Previous treatment
5. Change in the symptomatic treatment of mastocytosis or administration of any new treatment of mastocytosis within 4 weeks prior to baseline
6. Treatment with any investigational agent within 4 weeks prior to baseline
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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Olivier Lortholary, MD, PhD
Role: PRINCIPAL_INVESTIGATOR
Hôpital Necker, Paris, France
Locations
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UC Davis Health System , Department of Dermatology
Sacramento, California, United States
MD Anderson Cancer Centre
Houston, Texas, United States
CHU d'Amiens
Amiens, , France
Hôpital Avicenne
Bobigny, , France
CHU de Brest
Brest, , France
CHU de Caen
Caen, , France
CHU Clermont Ferrand
Clermont-Ferrand, , France
Hôpital Claude Huriez
Lille, , France
CHU Dupuytren
Limoges, , France
Hôpital Ambroise Paré
Marseille, , France
Hôpital Nord
Marseille, , France
Hôpital Central
Nancy, , France
CHU Hôtel Dieu
Nantes, , France
Hôpital l'Archet II
Nice, , France
Hôpital Necker
Paris, , France
Hôpital Tenon
Paris, , France
CHU Lyon Sud
Pierre-Bénite, , France
Centre Hospitalier Lyon Sud
Pierre-Bénite, , France
CHU Milétrie
Poitiers, , France
CHU Hôpital Sud
Rennes, , France
CHU de Saint-Etienne
Saint-Etienne, , France
Hôpital Purpan
Toulouse, , France
Hôpital Bretonneau
Tours, , France
Hôpital des Hauts Clos
Troyes, , France
Countries
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References
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Arock M. A new therapeutic advance for symptomatic systemic mastocytosis? Lancet. 2017 Feb 11;389(10069):576-578. doi: 10.1016/S0140-6736(16)31655-5. Epub 2017 Jan 7. No abstract available.
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.
Related Links
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Publication of results
Other Identifiers
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AB06006
Identifier Type: -
Identifier Source: org_study_id
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