Midostaurin in Indolent Systemic Mastocytosis

• Status: Unknown status
• Trial end date: 2015-05-01
• Target enrollment: 0
• Participant gender: All

Summary

Rationale: Patients with indolent or smoldering systemic mastocytosis can have severe disabling symptoms. Almost all patients have fatigue, a compromised quality of life, hampering normal functioning. Because this form of mastocytosis is not considered life-threatening, mast cell eradication has never been applied and patients receive only symptomatic therapy with histamine blockers. Midostaurin, a c-KIT inhibitor has shown activity regarding symptom control and decrease of malignant mast cells in patients with aggressive systemic mastocytosis (ASM) or mast cell leukemia

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: University Medical Center Groningen
• Treatments: 4'-N-benzoylstaurosporine; Midostaurin; Staurosporine

Criteria

Inclusion Criteria:

• Patients with Indolent Systemic Mastocytosis (ISM) or Smouldering Systemic Mastocytosis (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 form) with at least:

• a pre-study score of 4 or more on 3 non-related items,

• or a pre-study score of 5 or more on 2 non-related items.

• 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) for 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