E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Agressive Systemic Mastocytosis (ASM) or Mast Cell Leukemia (MCL) +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease (AHNMD) |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10056453 |
E.1.2 | Term | Aggressive systemic mastocytosis |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
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 |
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E.2.2 | Secondary objectives of the trial |
to evaluate:
- duration of response - time to response - overall survival - safety and tolerability of midostaturin - characterize KIT mutational status at baseline and after 6 cycles |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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 |
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E.4 | Principal 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
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E.5 End points |
E.5.1 | Primary end point(s) |
Overall response rate (ORR) |
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | Yes |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | Yes |
E.6.11 | Pharmacogenomic | Yes |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | No |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 14 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | Information not present in EudraCT |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
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E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 2 |