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

Phase 1

• Conditions: Aggressive Systemic Mastocytosis or Mast Cell Leukemia +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease; MedDRA version: 17.0Level: LLTClassification code 10056453Term: Aggressive systemic mastocytosisSystem Organ Class: 100000004851; Therapeutic area: Diseases [C] - Cancer [C04]

• Registration Number: EUCTR2008-000280-42-AT
• Lead Sponsor: ovartis Pharma Services AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2010-02-24
• Last Update Posted: 9 October 2017

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• Target Recruitment: 116

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
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range

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

Study & Design

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