Promising combo tackles FLT3 leukemia in early trial
NCT ID NCT03900949
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This early-phase study tested whether adding two targeted drugs, gemtuzumab ozogamicin and midostaurin, to standard chemotherapy is safe for people with a newly diagnosed, aggressive form of acute myeloid leukemia (AML) that has a FLT3 mutation. The trial enrolled 21 adults and aimed to find the best dose with acceptable side effects. The goal is to improve initial treatment for this hard-to-treat leukemia.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- gemtuzumab ozogamicin and midostaurin combined with standard chemotherapy (cytarabine and daunorubicin)
- What this could lead to
- If successful, this could lead to a more effective initial treatment for a specific type of AML, potentially improving remission rates.
- What could go wrong
- This is a very early (Phase I) safety study with only 21 participants. The combination may cause severe side effects and may not improve outcomes in larger trials.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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21 people
The number who actually took part.
- Started
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Mar 2019
- Finished
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Oct 2025
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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18 years and older
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * Ability to understand and the willingness to sign a written informed consent document * Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 * Newly diagnosed AML as confirmed by bone marrow and/or peripheral blood examination as indicated, with: * Confirmed CD33 positivity, per institutional standards * Presence of FLT3 internal tandem duplication (ITD) or tyrosine kinase domain (TKD) mutation as confirmed by next-generation sequencing (NGS) or other molecular method * Aspartate aminotransferase (AST) \< 2.5 x upper limit of normal (ULN; local laboratory) * Alanine aminotransferase (ALT) \< 2.5 x ULN * Total bilirubin \< 2 x ULN (except for patients with known Gilbert's syndrome) * Calculated creatinine clearance (according to the Cockcroft-Gault equation) \> 40 mL/min OR serum creatinine \< 1.5 x the ULN * Female patients of childbearing potential must agree to use adequate contraception (2 forms of contraception or abstinence) from the screening visit until 6 months following the last dose of study treatment. Should a woman become pregnant or suspect she is pregnant while she or her partner is participating in this study, she should inform her treating physician immediately * Male patients of childbearing potential having intercourse with females of childbearing potential must agree to abstain from heterosexual intercourse or have their partner use 2 forms of contraception from the screening visit until 4 months following the last dose of study treatment. They must also refrain from sperm donation from the screening visit until 4 months following the last dose of study treatment Exclusion Criteria: * Isolated myeloid sarcoma (meaning, patients must have blood or marrow involvement to enter study) * Acute promyelocytic leukemia (per World Health Organization classification) * Active central nervous system (CNS) involvement by AML, as assessed at discretion of principal investigator (PI) or treating physician and confirmed by lumbar puncture * Except for hydroxyurea, no other prior systemic anti-AML therapies may have been received prior to starting study therapy * Known history of veno-occlusive disease * Known active human immunodeficiency virus (HIV), active hepatitis B or active hepatitis C infection * Patients with the following will be excluded: uncontrolled intercurrent illness including, but not limited to, symptomatic congestive heart failure, unstable angina pectoris, serious cardiac arrhythmia, myocardial infarction within 6 months prior to enrollment, New York Heart Association (NYHA) class III or IV heart failure, severe uncontrolled ventricular arrhythmias * Patients with uncontrolled infection will not be enrolled until infection is treated * Any concurrent condition that, in the investigator's opinion, would jeopardize the safety of the patient or compliance with the protocol * Inability to take oral medication * Hypersensitivity to any study agent, or its excipients, when administered alone * Pregnancy or breastfeeding at the time of enrollment
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Ohio State University Comprehensive Cancer Center
Columbus, Ohio, 43210, United States
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Oregon Health and Science University Knight Cancer Institute - Northwest Portland
Portland, Oregon, 97210, United States
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