JOURNAL ONKOLOGIE – STUDIE

Masitinib in Severe Indolent or Smoldering Systemic Mastocytosis Unresponsive to Optimal Symptomatic Treatment

Studien-Informationen Einschlusskriterien Studien-Rationale Studien-Arme Geprüfte Regime

Rekrutierend

NCT-Nummer:
NCT04333108

Studienbeginn:
Juli 2020

Letztes Update:
06.05.2023

Wirkstoff:
Masitinib

Indikation (Clinical Trials):
Mastocytosis, Mastocytosis, Systemic

Geschlecht:
Alle

Altersgruppe:
Erwachsene (18+)

Phase:
Phase 3

Sponsor:
AB Science

Collaborator:
-

Studienleiter

Cristina Bulai Livideanu, MD, MSc
Principal Investigator
Centre Hospitalier Universitaire, Service de Dermatologie, Toulouse -France

Kontakt

Clinical Study Coordinator
Kontakt:
Phone: +33(0)147200014
E-Mail: clinical@ab-science.com» Kontaktdaten anzeigen

Studienlocations
(3 von 17)

University Hospital Charité
Berlin
(Berlin)
GermanyNoch nicht rekrutierend» Google-Maps

Centre Hospitalier Universitaire d'Amiens
Amiens
FranceRekrutierend» Google-Maps

Hospital Jean-Minjoz
Besançon
FranceRekrutierend» Google-Maps

Grenoble University Hospital
Grenoble
FranceRekrutierend» Google-Maps

Hospital Claude Huriez
Lille
FranceRekrutierend» Google-Maps

Marseille University Hospital Timone
Marseille
FranceRekrutierend» Google-Maps

Centre de référence de Mastocytose (CEREMAST)
Paris
FranceRekrutierend» Google-Maps

Poitiers University Hospital
Poitiers
FranceRekrutierend» Google-Maps

Centre Hospitalier Universitaire
Toulouse
FranceRekrutierend» Google-Maps

Erasmus University Medical Center
Rotterdam
NetherlandsRekrutierend» Google-Maps

Medical University of Gdańsk
Gdańsk
PolandRekrutierend» Google-Maps

The University Hospital in Krakow (Szpital Uniwersytecki w Krakowie)
Kraków
PolandRekrutierend» Google-Maps

University Hospital in Bucharest (Spitalul Universitar de Urgență București)
Bucharest
RomaniaRekrutierend» Google-Maps

Almazov National Medical Research Centre
Saint Petersburg
Russian FederationRekrutierend» Google-Maps

Dnipropetrovsk Clinical Association of Emergency Medical Care of Dnipropetrovsk Regional
Dnipropetrovs'k
UkraineRekrutierend» Google-Maps

Private Enterprise Private Manufacturing Company Acinus
Poltava
UkraineRekrutierend» Google-Maps

Guy's and St Thomas' NHS Foundation Trust
London
United KingdomRekrutierend» Google-Maps

Alle anzeigen

Studien-Informationen

Detailed Description:

Masitinib is a selective tyrosine kinase inhibitor that modulates mast cell activity via

inhibition of c-Kit, Lyn and Fyn kinase signaling pathways. This is a multicenter,

randomized, double-blind, placebo-controlled, 2-parallel-group, trial comparing oral

masitinib versus placebo in the treatment of patients suffering from smouldering or indolent

systemic mastocytosis with severe symptoms of mast cell mediator release (also referred to as

handicaps), unresponsive to optimal symptomatic treatment. The treatment period is 24 weeks.

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.

Ein-/Ausschlusskriterien

Inclusion Criteria:

1. Patient with one of the following documented mastocytosis subtypes (variants):

Smouldering Systemic Mastocytosis, Indolent Systemic Mastocytosis

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:

1. Patient with one of the following mastocytosis: Cutaneous Mastocytosis, Systemic

Mastocytosis with an Associated clonal Hematologic Non Mast cell lineage Disease

(SM-AHNMD), Mast cell leukemia (MCL), Aggressive systemic mastocytosis (ASM)

2. Previous treatment with any Tyrosine Kinase Inhibitor

3. Treatment with any investigational agent within 8 weeks prior to screening.

Studien-Rationale

Primary outcome:

1. Cumulative response (3R75%) (Time Frame - 24 weeks):
Cumulative response in at least one of three severe baseline symptoms of mast cell mediator release (pruritus, flushes, or depression). Response was defined as a 75% improvement from baseline for any of these three symptoms. Cumulative response was defined as the number of actual responses between weeks 8 and 24, divided by the total number of possible responses over the same treatment period (ie, with five scheduled visits, each patient had a maximum of five to 15 possible responses depending on the number of severe baseline symptoms).

Secondary outcome:

1. Cumulative response (4R75%) (Time Frame - 24 weeks):
Cumulative response in at least one of four severe baseline symptoms of mast cell mediator release (pruritus, flushes, depression, or asthenia). Response is defined as a 75% improvement from baseline for any of these four symptoms. Cumulative response was defined as the number of actual responses between weeks 8 and 24, divided by the total number of possible responses over the same treatment period (ie, with five scheduled visits, each patient had a maximum of five to 20 possible responses depending on the number of severe baseline symptoms).

2. Cumulative response (2R75%) (Time Frame - 24 weeks):
Cumulative response in at least one of two severe baseline symptoms of mast cell mediator release (pruritus or flushes). Response is defined as a 75% improvement from baseline for either of these two symptoms. Cumulative response is defined as the number of actual responses between weeks 8 and 24, divided by the total number of possible responses over the same treatment period (ie, with five scheduled visits, each patient had a maximum of five to 10 possible responses depending on the number of severe baseline symptoms).

3. Cumulative response (Time Frame - 24 weeks):
Cumulative response on each of the individual handicaps. Response is defined as a 75% improvement from baseline for either of these two symptoms. Cumulative response is defined as the number of actual responses between weeks 8 and 24, divided by the total number of possible responses over the same treatment period.

Studien-Arme

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

Geprüfte Regime

Masitinib (AB1010):
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.

Quelle: ClinicalTrials.gov

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