This trial is active, not recruiting.

Condition leukemia
Treatment midostaurin (pkc412)
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


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

Intervention model single group assignment
Primary purpose treatment
Masking no masking
Midostaurin was administered at a dose of 100 mg twice daily (bid) in continuous cycles of 28 days until disease progression, intolerable toxicity or withdrawal due to any cause, whichever occurred first.
midostaurin (pkc412)
Midostaurin was provided as 25 mg soft gelatin capsules for oral administration.

Primary Outcomes

Overall response rate (ORR)
time frame: 6 months

Secondary Outcomes

Duration of response
time frame: 5 years
Time to response
time frame: 5 years
Overall survival (OS)
time frame: 5 years

Eligibility Criteria

All participants at least 18 years old.

Key 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 Key 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

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 March 2017.
Information provided to ClinicalTrials.gov by Novartis.