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

Phase 1

Active, not recruiting

• Conditions: Agressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease (AHNMD); MedDRA version: 9.1Level: LLTClassification code 10056453Term: Aggressive systemic mastocytosis

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

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2008-07-25
• Last Update Posted: 13 November 2017

Recruitment & Eligibility

• Status: Not Recruiting
• Sex: All
• Target Recruitment: 60

Inclusion Criteria

- Age > 18 and = 70 yearsof age
- ECOG performance status of 0-3
- life expectancy > 12 weeks
- ECG QTc interval < 450 ms
-meeting 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
- diagnose of ASM or MCL according to WHO criteria for SM plus the established
criteria for ASM or MCL
- informed consent
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 theprotocol
- any other concurrent severe known disease and/or severe uncontrolled medical
condiction which could compromise participation in teh study
- confirmed diagnosis of HIV infection or active viral hepatitis
- female patients who are pregnant or breast feeding or adults of reproductive
potential not employing an effective method of birth control
- patients who have demonstrated relapse to more than 2 prior regimen of SM
treatment

Study & Design

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

Primary Outcome Measures

Name	Time	Method
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:<br><br>- duration of response<br>- time to response<br>- overall survival<br>- safety and tolerability of midostaturin<br>- characterize KIT mutational status at baseline and after 6 cycles;Primary end point(s): Overall response rate (ORR)