A phase II study to determine if the drug midostaurin taken orally twice daily is effective and safe in treating patients with Agressive Systemic Mastocytosis or Mast Cell Leukemia +/- an associated hematological clonal non-mast cell lineage disease

A single arm, Phase II, Open-label Study to determine the efficacy of 100 mg twice daily oral dosing of midostaurin administered to patients with Agressive Systemic Mastocytosis or Mast Cell Leukemia +/- an associated hematological clonal non-mast cell lineage disease - CPKC412D2201

Status

Active, not recruiting

Phases

Phase 2

Study type

Interventional

Source

EU CTR

Registry ID

EUCTR2008-000280-42-BE

Enrollment

Registered

2008-07-17

Start date

2008-08-07

Completion date

Unknown

Last updated

2017-10-02

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

Conditions

Agressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease (AHNMD) MedDRA version: 17.0 Level: LLT Classification code 10056453 Term: Aggressive systemic mastocytosis System Organ Class: 100000004851

Interventions

Product Name: midostaurin (INN) Product Code: PKC412 Pharmaceutical Form: Capsule, soft INN or Proposed INN: midostaurin CAS Number: 120685-11-2 Current Sponsor code: PKC412 Concentration unit: mg mil

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: - Male or female pts aged = 18 years of age - ECOG performance status of 0-3 - life expectancy > 12 weeks - ECG QTc interval = 450 ms - following lab values: AST and ALT =2.5 x ULN, if caused by ASM/MCL: = 5 x ULN Serum Bilirubin = 1.5 ULN, if related to ASM/MCL: =3x ULN Serum Creatinine = 2.0 mg/dl - diagnosed with ASM or MCL according to WHO criteria for SM plus for ASM or MCL 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 40 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 40

Exclusion criteria

Exclusion criteria: - patients unwilling or unable to comply with the protocol - any other concurrent severe known disease and/or severe uncontrolled medical condiction which could compromise participation in the study - patients with cardiovascular disease including congestive heart failure grade III or IV according to the NYHA classification, left ventricular ejection fraction of <50%, myocardial infection within previous 6 months and poorly controlled hypertension - confirmed diagnosis of HIV infection or active viral hepatitis - female patients who are pregnant or breast feeding or adults of reproductive potential not employing a highly effective method of birth control which is defined as a birth control which results in a low failure rate (i.e. less than 1% per year) when used consistently and correctly - patients presenting with an AHNMD requiring immediate cytoreductive therapy or targeted drugs (other than midostaurin) - patients who have demonstrated relapse to 3 or more prior regimens of SM treatment

Design outcomes

Primary

Measure	Time frame
Main Objective: To determine the efficacy of midostaurin in patients with ASM or MCL with or without AHNMD when administered orally at a dose of 100 mg b.i.d. continuously for 6 cycles (of 4 weeks each) as measured by overall response rate;Secondary Objective: to evaluate: - duration of response - time to response - overall survival - progression free survival - safety and tolerability of midostaturin to characterize KIT mutational status in the SM compartment and if applicabe also in the AHNMD compartment of the disease at baseline, after 6 cycles of therapy and at end of study (EOS) and explore potential association with efficacy outcomes. To evaluate histopathologic response based on mast cell infiltration in the bone marrow (BM) and changes in serum tryptase levels as surrogate marker for histopathologic response;Primary end point(s): Overall response rate (ORR);Timepoint(s) of evaluation of this end point: After continuously treatment of 6 cycles (of 4 weeks each), response must be confirmed 8 weeks apart.	—

Measure

Time frame

Main Objective: To determine the efficacy of midostaurin in patients with ASM or MCL with or without AHNMD when administered orally at a dose of 100 mg b.i.d. continuously for 6 cycles (of 4 weeks each) as measured by overall response rate;Secondary Objective: to evaluate: - duration of response - time to response - overall survival - progression free survival - safety and tolerability of midostaturin to characterize KIT mutational status in the SM compartment and if applicabe also in the AHNMD compartment of the disease at baseline, after 6 cycles of therapy and at end of study (EOS) and explore potential association with efficacy outcomes. To evaluate histopathologic response based on mast cell infiltration in the bone marrow (BM) and changes in serum tryptase levels as surrogate marker for histopathologic response;Primary end point(s): Overall response rate (ORR);Timepoint(s) of evaluation of this end point: After continuously treatment of 6 cycles (of 4 weeks each), response must be confirmed 8 weeks apart.

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Secondary

Measure	Time frame
Secondary end point(s): -duration of response - event free survival - time to response - overall survival - safety - histopathologic response;Timepoint(s) of evaluation of this end point: same as primary endpoint	—

Countries

Australia, Austria, Belgium, Canada, France, Germany, Italy, Netherlands, Norway, Poland, United Kingdom, United States

Contacts

Public ContactClinical Trial Information Desk

Novartis Pharma AG

clinicaltrial.enquiries@novartis.com+41 61 324 1111

Outcome results

None listed

Source: EU CTR (via WHO ICTRP) · Data processed: Feb 4, 2026