Study to Assess the Safety and Efficacy of Midostaurin (PKC412) in Combination With Standard Chemotherapy During Induction and Consolidation Followed by 12 Months of Monotherapy in Patients With Newly-diagnosed FLT3-mutated Acute Myeloid Leukemia.
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|ClinicalTrials.gov Identifier: NCT03379727|
Recruitment Status : Not yet recruiting
First Posted : December 20, 2017
Last Update Posted : December 20, 2017
|Condition or disease||Intervention/treatment||Phase|
|Acute Myeloid Leukemia||Drug: Midostaurin||Phase 3|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||300 participants|
|Intervention Model:||Single Group Assignment|
|Intervention Model Description:||
Considering the limited safety impact and the significant clinical benefit of the addition of midostaurin to the standard "7+3" regimen in the RATIFY study (CPKC412A2301), this Phase IIIb study is designed as a single arm study and allows the assessment of variation of the "7+3" regimen in an extended patient population compared to RATIFY (higher dose of daunorubicin (60-90 mg/m2/day), the substitution of daunorubicin by idarubicin (12mg/m2/day), and lower dose of cytarabine (100-200 mg/m2/day) and the "5+2" reduced dose regimen). Safety is the primary endpoint. CR/CRi (see Section 7.2.1) in induction, consolidation and maintenance therapy is collected as secondary endpoint.
Patients who are newly diagnosed with AML, have a known FLT3 ITD or TKD, mutation and have recently started on "7+3" or "5+2" in induction and high dose of cytarabine in consolidation will be consented and screened for the clinical study.
Number of Arms:
|Masking:||None (Open Label)|
|Official Title:||An Open-label, Multi-Center, Phase IIIb Study to Assess the Safety and Efficacy of Midostaurin (PKC412) in Patients 18 Years of Age or Older With Newly-diagnosed FLT3-mutated Acute Myeloid Leukemia Who Are Eligible for "7+3" or "5+2" Chemotherapy|
|Estimated Study Start Date :||December 31, 2017|
|Estimated Primary Completion Date :||August 15, 2021|
|Estimated Study Completion Date :||August 15, 2021|
Induction phase - D8 to D28 in combination with standard of care (7+3 or 5+2 chemotherapy) up to 2 cycles Consolidation phase - D8 to D28 in combination with cytarabine up to 4 cycles Maintenance phase - D1 to D28 up to 12 cycles
Orally administered inhibitor of multiple tyrosine kinases
- Percentage of patients with AEs, Grade 3&4 AEs, SAEs, AEs leading to discontinuation, and deaths up to 24 months. [ Time Frame: Baseline up to approximatly 24 months ]To further assess the safety of midostaurin in induction, consolidation and maintenance therapy, including, the "7+3" regimen, higher dose of Daunorubicin (60-90mg/m2/day), the substitution of Daunorubicin by Idarubicin and lower dose of Cytarabine (100-200 mg/m2/day) and also allowing the "5+2" reduced dose regimen.
- Percentage of patients with CR/CRi as per local assessment [ Time Frame: Baseline up to approximately 24 months ]CR/CRi rate is defined as the percentage of patients with complete remission (CR) or complete remission with incomplete hematologic recovery (CRi) as per local assessment, in induction, consolidation and maintenance phase. CR/CRi rate will be calculated based on the full analysis set (FAS).
- Health Care Resource Utilization during maintenance [ Time Frame: During maintenance up to 12 months ]Collection of health care resource utilization (HCRU) data will focus on hospitalization: reason for the hospitalization, number of hospital days by ward type (e.g. hospital unit, emergency room, intensive care unit), discharge status, and the names of concomitant medications during hospital stay. These measures will be used to quantify the number of hospital day's impact of therapy during the maintenance phase and derive components of the economic impact of midostaurin during maintenance.
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03379727
|Contact: Novartis Pharmaceuticals||+41613241111||Novartis.email@example.com|
|Contact: Novartis Pharmaceuticals|