Overview

This trial is active, not recruiting.

Condition leukemia
Treatment midostaurin
Phase phase 2
Target FLT-3
Sponsor Novartis Pharmaceuticals
Start date October 2008
End date July 2017
Trial size 116 participants
Trial identifier NCT00782067, 2008-000280-42, CPKC412D2201

Summary

This study will investigate if the drug midostaurin taken orally twice daily is effective and safe in treating patients with aggressive systemic mastocytosis or mast cell leukemia with or without an additional hematological neoplasm.

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Endpoint classification safety/efficacy study
Intervention model single group assignment
Masking open label
Primary purpose treatment
Arm
(Experimental)
midostaurin

Primary Outcomes

Measure
Overall response rate according to established criteria by assessing clinical findings at the end of 6 cycles
time frame: at the end of 6 cycles

Secondary Outcomes

Measure
Duration of response
time frame: throughout the study
Time to response
time frame: throughout the study
Adverse event rate
time frame: throughout the study
Overall survival (OS)
time frame: throughout the study
KIT mutational status at diagnosis and after 6 cycles of therapy
time frame: at diagnosis and after 6 cycles of therapy

Eligibility Criteria

Male or female participants at least 18 years old.

Inclusion Criteria: - Confirmed diagnosis of aggressive systemic mastocytosis (ASM) or mast cell leukemia (MCL) according to WHO criteria for SM and established criteria for ASM and MCL (Valent et al 2003), presenting with at least one measurable C-Finding. - ECOG performance status of 0-3 - Life expectancy > 12 weeks - ECG: QTc interval ≤ 450 ms - Meeting the following laboratory values: - AST and ALT must be ≤ 5 x Upper Limit of Normal (ULN) if this elevation is solely due to ASM/MCL, otherwise AST, ALT must be ≤ 2.5 x ULN - Serum Bilirubin must be ≤ 3 x Upper Limit of Normal (ULN) if this elevation is solely due to ASM/MCL, otherwise serum bilirubin must be - 1.5 x ULN - Serum Creatinine ≤ 2.0 mg/dL Exclusion Criteria: - Any other concurrent severe known disease (except carcinoma in-situ) concurrent severe and/or uncontrolled medical condition including congestive heart failure grade III or IV according to the NYHA classification or with ejection fraction < 50%, etc. - Patients with any pulmonary infiltrate including those suspected to be of infectious origin. Exception: Patients with a pleural effusion related to the disease under study as confirmed by the investigator are permitted to enter the study - Patients who have demonstrated relapse to 3 or more prior regimens of SM treatment (not including those given for supportive care) - Patients who have received any investigational agent, chemotherapy, interferon-α, or 2-chlorodeoxyadenosine (2-CdA, cladribine) within 30 days prior to first dose - Patients who have ASM with eosinophilia and known positivity for the FIP1L1-PDGFRα fusion unless they have demonstrated relapse or disease progression on prior imatinib therapy Other protocol-defined inclusion/exclusion criteria may apply

Additional Information

Official title A Single Arm, Phase II, Open-Label Study to Determine the Efficacy of 100mg Twice Daily Oral Dosing of Midostaurin Administered to Patients With Aggressive Systemic Mastocytosis or Mast Cell Leukemia +/- an Associated Hematological Clonal Non-Mast Cell Lineage Disease
Trial information was received from ClinicalTrials.gov and was last updated in January 2016.
Information provided to ClinicalTrials.gov by Novartis.