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

Phase 2

Completed

Conditions: mastocytosis
mast cell disease
10018865

Registration Number: NL-OMON39686

Lead Sponsor: niversitair Medisch Centrum Groningen

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Completed

Sex: Not specified

Target Recruitment: 20

Inclusion Criteria

* Patients with ISM or SSM according to the WHO criteria
* Presence of the D816V c-KIT mutation
* Serum tryptase > 20 mg/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

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

Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Percent change in the Sumscore of all symptoms assessed by the Mastocytosis<br /><br>Symptom Assessment Form (MSAF) after 12 weeks. </p><br>

Secondary Outcome Measures

Name	Time	Method
<p>Persistence of improvement symptom score at 6 months. Percent change in the<br /><br>mast cell burden (bone marrow infiltrate, skin infiltrate, serum tryptase<br /><br>levels) after 6 months. Number and grading of CTC adverse events during the 6<br /><br>months of therapy. </p><br>