Clinical trial · Interventional
A Study of the Safety and Preliminary Efficacy of Oral Midostaurin (PKC412) in Relapsed or Refractory Pediatric Leukemia
A Phase I/II, Open-label, Dose-escalating Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Twice Daily Oral Midostaurin and to Evaluate the Preliminary Clinical and Pharmacodynamic Response in Pediatric Patients With Relapsed or Refractory Leukemia
- Source
- ClinicalTrials.gov
- Retrieved
- Sep 8, 2026
- Layer
- normalized (units and labels harmonized; values unchanged)
- Run
- ING-CLINICALTRIALS-20260908-000001
Why stopped (as posted): Despite considerable efforts to boost recruitment during the final year of the study, no new patients were enrolled.
Summary
Brief summary (as posted)
This is a phase I/II pediatric dose-ranging study that will evaluate the safety, tolerability, clinical response, pharmacokinetics and pharmacodynamics of midostaurin in patients \<18 years of age who have relapsed or refractory acute leukemias that may benefit from administration of midostaurin, including MLL-rearranged ALL and FLT3 positive AML.
Conditions
Conditions (2)
Free-text conditions as registered, with the CancerIndex entity they were reconciled to and the match type.
| Condition (as posted) | Mapped entity | Match | Confidence |
|---|---|---|---|
| Acute Lymphoblastic Leukemia | Acute Lymphoblastic Leukemia | ONTOLOGY_EXACT | 0.98 |
| Acute Myeloid Leukemia | Acute Myeloid Leukemia | CURATED_BROADER | 0.80 |
Interventions
Interventions (1)
| Intervention | Type | Mapped drug | Match |
|---|---|---|---|
| midostaurin | Drug | Midostaurin | ALIAS |
Design
Arms and outcomes
Arms (2)
- type
- EXPERIMENTAL
- label
- 30 mg/m^2 bid
- description
- Participants received bodyweight and body surface area (BSA) stratified dose of midostaurin 30 mg/m\^2 twice daily (bid) through oral route. The total daily dose in 30 mg/m\^2 bid cohort was 60 mg/m\^2.
- interventionNames
- Drug: midostaurin
- type
- EXPERIMENTAL
- label
- 60 mg/m^2 bid
- description
- Participants received bodyweight and BSA stratified dose of midostaurin 60 mg/m\^2 bid through oral route. The total daily dose in 60 mg/m\^2 bid cohort was 120 mg/m\^2.
- interventionNames
- Drug: midostaurin
Primary outcomes (1)
- measure
- Maximum Tolerated Dose (MTD) of Midostaurin- Posterior Probability of DLT
Eligibility
Eligibility (as posted)
- Sex
- All
- Minimum age
- 3 Months
- Maximum age
- 18 Years
Show eligibility criteria text
Inclusion Criteria: * Mixed-lineage leukemia (MLL) gene rearranged Acute Lymphoblastic Leukemia (ALL), that does not respond to treatment or has relapsed from prior treatment; or FLT3 mutated Acute Myeloid Leukemia (AML) that does not respond to a second treatment or has relapsed from 2 prior treatments * Normal organ function, and chest x-ray * Expected survival greater than 8 weeks * Can care for most of personal needs and perform at least minimum activity Exclusion Criteria: * Patients with symptomatic leukemic central nervous system involvement or isolated extramedullary leukemia * Patients must not have received other treatments for leukemia within a predefined time period, 72 hours for medications, 2 months for transplants * Patients with heart function that is not normal * Patients with HIV or hepatitis * Patients with another severe disease or medical condition besides leukemia Other protocol-defined inclusion/exclusion criteria may apply
References
Publications (0)
Data not yet available