A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and P… (NCT03591510) | Clinical Trial Compass
Active — Not RecruitingPhase 2
A Global Study of Midostaurin in Combination With Chemotherapy to Evaluate Safety, Efficacy and Pharmacokinetics in Newly Diagnosed Pediatric Patients With FLT3 Mutated AML
This study will evaluate the safety, efficacy and pharmacokinetics of midostaurin in combination with standard chemotherapy in pediatrics patients with newly diagnosed FLT3-mutated Acute Myeloid Leukemia. The study has two parts: Part 1 to define the Recommended Phase 2 Dose, and Part 2 to evaluate safety and tolerability and efficacy of midostaurin. Both parts will consist of 2 induction blocks, 3 consolidation blocks, 12 cycles of post-consolidation consisting of continuous therapy with midostaurin, and a follow-up phase.
Who can participate
Age range
3 Months – 17 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Documented Diagnosis of previously untreated de novo AML according to WHO 2016 criteria
* Presence of a FLT3 mutation status as measured/confirmed by a designated lab with results available prior first dose of Midostaurin
* Patients with Lansky or Karnofsky performance status equal or superior to 60
* Patient with the following laboratory value : AST and ALT ≤ 3times ULN
* Serum Total bilirubin ≤ 1.5times ULN
* Estimated creatinine clearance ≥30ml/min
Exclusion Criteria:
* Any concurrent malignancy, AML with Philadelphia Chromosome, AML-DS, JMML
* Symptomatic leukemic CNS involvement
* Isolated extramedullary leukemia, secondary AML and MDS
* Acute Promyelocytic Leukemia with the PML RARA rearrangement
* Patient who have received prior treatment with a FLT3 inhibitor. However, up to 1 week of FLT3 inhibitor (except midostaurin) exposure prior to study enrollment is permissible.
Other protocol-defined inclusion/exclusion criteria may apply
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Part 1 of the study: Occurence of dose limiting toxicities (DLT)
Timeframe: From the start of midostaurin treatment in Block 1 to the end of Block 2, from Day 1 to Day 84
2
Part 2 of the study: To evaluate Safety of midostaurin (30mg/m2 bid or 1 mg/kg bid for participants <10 kg body weight) in sequential combination with chemotherapy followed by 12 cycles of midostaurin post-consolidation therapy.
Timeframe: From the start of treatment up to 5 years follow-up of last patient
3
Part 2 of the study: To evaluate Tolerability of midostaurin (30mg/m2 bid or 1 mg/kg bid for participants <10 kg body weight) in sequential combination with chemotherapy followed by 12 cycles of midostaurin post-consolidation therapy.
Timeframe: From the start of treatment up to 5 years follow-up of last patient