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 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

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

NL-OMON

Registry ID

NL-OMON39686

Enrollment

Registered

2013-01-31

Start date

2013-09-13

Completion date

Unknown

Last updated

2024-04-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

mastocytosis

Interventions

treatment with Midostaurin, twice daily 100 mg orally for 6 months continuously.

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

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

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

Design outcomes

Primary

Measure	Time frame
Percent change in the Sumscore of all symptoms assessed by the Mastocytosis Symptom Assessment Form (MSAF) after 12 weeks.	—

Secondary

Measure	Time frame
Persistence of improvement symptom score at 6 months. Percent change in the mast cell burden (bone marrow infiltrate, skin infiltrate, serum tryptase levels) after 6 months. Number and grading of CTC adverse events during the 6 months of therapy.	—

Countries

Netherlands

Outcome results

None listed

Source: NL-OMON (via WHO ICTRP)