Midostaurin in Indolent Systemic Mastocytosis

• Conditions: Indolent or smoldering mastocytosis; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2012-004868-22-NL
• Lead Sponsor: niversity Medical Center Groningen

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2013-07-09
• Last Update Posted: 10 February 2014

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

? 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

Exclusion Criteria

- 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

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified