Phase II single arm open pilot study to demonstrate the efficacy of midostaurin in symptom improvement and decrease of mast cell burden in patients with indolent or smoldering systemic mastocytosis - Midostaurin in Indolent Systemic Mastocytosis

niversity Medical Center Groningen0 个研究点开始时间: 2013年7月9日最近更新: 2014年2月10日

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•? Patients with ISM or SSM according to the WHO criteria
•? Presence of the D816V c-KIT mutation
•? Serum tryptase =20 µg/l
•? Serious mediator-related symptoms that cannot be controlled by H1 and H2 blocking drugs. Symptoms will be scored by an adapted MSAF (mastocytosis symptom assessment form15) with at least
•o a pre-study score of 4 or more on 3 non-related items,
•o or a pre-study score of 5 or more on 2 non-related items.
•o one item from the scoring list can be replaced by flushes 7 or more per week or anaphylactic attacks 1 or more per week
•? Age >18 years
•? Willingness to apply optimal contraceptive measures (double barrier method, both men and women) if applicable, e.g. women below the age of 55, men at all ages; for both: if sexually active.
•? Written informed consent
•Are the trial subjects under 18? no
•Number of subjects for this age range:
•F.1.2 Adults (18-64 years) yes
•F.1.2.1 Number of subjects for this age range 20
•F.1.3 Elderly (>=65 years) no
•F.1.3.1 Number of subjects for this age range

•- Aggressive systemic mastocytosis, mast cell leukemia, or ASM with or without
•accompanying non-clonal related non-mast cell disorder (SM-ANHMD).
•? Any known other present malignancy, non-melanoma skin cancers excluded
•? History of malignancy within the last 5 years, non-melanoma skin cancers excluded
•? Any serious comorbidity interfering with therapy compliance and follow-up compliance
•? Pregnancy
•? Patients not willing or who are not able to comply with contraceptive measures

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