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Protein Kinase Inhibitor

Midostaurin + Chemotherapy for Acute Myeloid Leukemia

Phase 2

Recruiting

Research Sponsored by Novartis Pharmaceuticals

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

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

Must not have

Any concurrent malignancy, AML with Philadelphia Chromosome, AML-DS, JMML

Isolated extramedullary leukemia, secondary AML and MDS

Timeline

Screening 3 weeks

Treatment Varies

Follow Up from the start of treatment up to 5 years follow-up of last patient

Awards & highlights

No Placebo-Only Group

Summary

This trial will study the safety and effectiveness of midostaurin when combined with standard chemotherapy in children with a specific type of leukemia. The study will be done in two parts and will involve giving the drug to children over a period of several months.

Who is the study for?

This trial is for pediatric patients with newly diagnosed FLT3-mutated Acute Myeloid Leukemia. They must have a certain level of physical function, normal liver and kidney tests, and no prior treatment with FLT3 inhibitors or other cancers. Patients with specific types of AML or leukemia in the brain are excluded.

What is being tested?

The study tests Midostaurin combined with standard chemotherapy to see its safety, how well it works, and how the body processes it in children with AML. It includes two phases: one to find the best dose and another to assess long-term effects over several treatment cycles followed by continuous therapy.

What are the potential side effects?

Midostaurin may cause side effects like nausea, vomiting, diarrhea, weakness, headache, mouth sores, rash or allergic reactions. Chemotherapy can lead to hair loss, infection risk increase due to low blood cell counts, bleeding issues from low platelets and potential damage to organs.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I have a new diagnosis of AML according to the latest standards.

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My lab tests show a FLT3 mutation.

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My kidneys are working well enough (creatinine clearance ≥ 30 ml/min).

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I can care for myself but may need occasional help.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I do not have any other cancer types or specific leukemia conditions.

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My condition is a type of leukemia that affects areas outside the bone marrow, or I have secondary AML or MDS.

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My leukemia is of a specific type known as Acute Promyelocytic.

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I have symptoms from leukemia affecting my brain or spinal cord.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ from the start of treatment up to 5 years follow-up of last patient

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~from the start of treatment up to 5 years follow-up of last patient

This trial's timeline: 3 weeks for screening, Varies for treatment, and from the start of treatment up to 5 years follow-up of last patient for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Part 1 of the study: Occurence of dose limiting toxicities (DLT)

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.

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.

Secondary study objectives

Part 1 and Part 2 of the study: Plasma concentrations of midostaurin and its metabolites

Part 2 of the study: Disease free survival (DFS)

Part 2 of the study: Event Free Survival (EFS)

+6 more

Side effects data

From 2021 Phase 3 trial • 301 Patients • NCT03379727

19%

Nausea

13%

Neutropenia

Diarrhoea

Alanine aminotransferase increased

Hypertension

Thrombocytopenia

Anaemia

Vomiting

Oedema peripheral

Arthralgia

Pyrexia

Rash

Platelet count decreased

Headache

Back pain

Aspartate aminotransferase increased

Constipation

Asthenia

Cough

Anxiety

Pneumonia

Leukocytosis

Subileus

Hypokalaemia

Abdominal pain upper

Pain in extremity

Abdominal pain

Electrocardiogram QT prolonged

Musculoskeletal chest pain

Oral herpes

Prostate cancer

Insomnia

Large intestinal obstruction

Endocarditis

Hypotension

100%

80%

60%

40%

20%

Study treatment Arm

Maintenance Phaase

Induction Phase

Consolidation Phase

Overall

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Chemotherapy followed by MidostaurinExperimental Treatment6 Interventions

In Part 1, midostaurin with standard induction (Block 1 induction according to local practice, Block 2 induction containing fludarabine, cytarabine, daunorubicin/idarubicin) and consolidation (Block 3: cytarabine + mitoxantrone, Block 4: cytarabine + etoposide, Block 5: cytarabine) followed by single agent midostaurin post-consolidation therapy. In Part 2, midostaurin with standard induction (Block 1 induction according to local practice, Block 2 induction containing cytarabine + mitoxantrone) and consolidation (Block 3: cytarabine + etoposide, Block 4: cytarabine + mitoxantrone, Block 5: cytarabine) followed by single agent midostaurin post-consolidation therapy.

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Midostaurin

2018

Completed Phase 3

~1640

Fludarabine

2012

Completed Phase 4

~1860