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Phase 1b Study of SNDX-5613 for Acute Myeloid Leukemia

This study is testing a new drug, SNDX-5613, in combination with standard chemotherapy drugs, daunorubicin and cytarabine, for people newly diagnosed with acute myeloid leukemia (AML). Specifically, it's for those whose AML has certain genetic changes (NPM1 mutated/FLT3 wildtype or MLL/KMT2A rearranged or NUP98 alterations). SNDX-5613 works by blocking signals inside cancer cells that they need to survive. Researchers want to find the safest and most effective dose of SNDX-5613 when added to standard chemotherapy. The goal is to see if this combination can shrink or stabilize the cancer better than chemotherapy alone. You would be between 18 and 75 years old to participate.

Study design
This is a Phase 1b interventional study, meaning it's an early-stage study to find the right dose and check for safety. It plans to enroll 38 participants.
What's involved
You would undergo blood collection, bone marrow aspiration, and bone marrow biopsy. You would also receive the study drugs daunorubicin, cytarabine, and SNDX-5613.
Compensation
Not stated in the trial record.
Follow-up
The study measures outcomes from the start of treatment up to 42 days after induction or re-induction, and up to 42 days after consolidation or until recovery from treatment side effects.

AI-generated from the public study record. Only the study team can confirm whether you're eligible — confirm details with them before making decisions.

NCT05886049

A Phase 1b Study of Menin Inhibitor SNDX- 5613 in Combination With Daunorubicin and Cytarabine in Newly Diagnosed Patients With Acute Myeloid Leukemia and NPM1 Mutated/FLT3 Wildtype or MLL/KMT2A Rearranged or NUP98 Alterations Disease

Recruiting
PHASE1Ages 18–75InterventionalTreatment
National Cancer Institute (NCI)
~38 participants
Updated 2026-08-25 on ClinicalTrials.gov
What's tested:Biospecimen CollectionBone Marrow AspirationBone Marrow BiopsyCytarabineDaunorubicinMultigated Acquisition Scan

At a glance

Recruiting sites
21 of 23 listed sites are recruiting right now
RecruitingSuspended, closed, or not yet open
What they're measuring
Recommended dose for expansion (RDE) for Induction
Measured over From day 1 to 42 of Induction or Re-Induction
+2 more outcomes measured

Conditions

Acute Myeloid Leukemia
Acute Myeloid Leukemia With KMT2A Rearrangement
Acute Myeloid Leukemia With NPM1 Mutation
Secondary Acute Myeloid Leukemia

Explore related trials

Acute Myeloid LeukemiaAcute Myeloid Leukemia with KMT2A RearrangementAcute Myeloid Leukemia with NPM1 MutationSecondary Acute Myeloid LeukemiaCD33 GeneKMT2A GeneMeninNPM1 GeneNUP98 GeneReceptor-Type Tyrosine-Protein Kinase FLT3

Where it's being run

23 sites across 11 states
Florida5
Kansas4
North Carolina3
Ohio3
California2
Illinois1
Maryland1
Massachusetts1

Study leadership

  • Alice S Mims · PRINCIPAL_INVESTIGATOR · Ohio State University Comprehensive Cancer Center LAO

Who to contact

Site Public Contact
[email protected]
877-827-8839
Email the study team

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Do you actually qualify for this trial?

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Eligibility criteria

Inclusion

Dose escalation: Patients ages 18-75 years at time of diagnosis with NPM1-mutated/FLT3-ITD wildtype and NPM1-mutated/FLT3-TKD wildtype with high-risk features (adverse risk genetics per European LeukemiaNet \[ELN\] 2022 criteria, age ≥ 60 years, or secondary AML defined as either arising from a prior hematological malignancy or therapy-related), MLL (KMT2A) rearranged or NUP98 altered, untreated AML and who are candidates for intensive induction chemotherapy. Patients with CD33+ AML are eligible for this protocol.
Dose expansion: Patients ages 18-75 years at time of diagnosis with NPM1-mutated/FLT3-ITD wildtype and NPM1-mutated/FLT3-TKD wildtype (any patient-does not require high-risk features), MLL (KMT2A) rearranged, or NUP98 altered, untreated AML and who are candidates for intensive induction chemotherapy. Patients with CD33+ AML are eligible for this protocol
Because no dosing or adverse event data are currently available on the use of SNDX-5613 in combination with daunorubicin and cytarabine in patients \< 18 years of age, children are excluded from this study
Eastern Cooperative Oncology Group (ECOG) performance status =\< 2 (Karnofsky \>= 60%). Patients over the age of 65 must have an ECOG performance status of 0-1
Total bilirubin ≤ 1.5 x institutional upper limit of normal (ULN), except for patients with Gilbert's syndrome where required to be ≤ 3 x institutional ULN
Aspartate aminotransferase (AST)(serum glutamic oxaloacetic transaminase \[SGOT\])/alanine aminotransferase (ALT)(serum glutamic pyruvate transaminase \[SGPT\]) =\< 3 x institutional upper limit of normal (ULN)
Glomerular filtration rate (GFR) \>= 60 mL/min/1.73 m\^2 (via the Chronic Kidney Disease Epidemiology \[CKD-EPI\] glomerular filtration rate estimation)
Human immunodeficiency virus (HIV)-infected patients on effective anti-retroviral therapy with undetectable viral load within 6 months are eligible for this trial
For patients with evidence of chronic hepatitis B virus (HBV) infection, the HBV viral load must be undetectable on suppressive therapy, if indicated
Patients with a history of hepatitis C virus (HCV) infection must have been treated and cured. For patients with HCV infection who are currently on treatment, they are eligible if they have an undetectable HCV viral load
Patients with a prior or concurrent malignancy whose natural history or treatment does not have the potential to interfere with the safety or efficacy assessment of the investigational regimen are eligible for this trial
Patients with known history or current symptoms of cardiac disease, or history of treatment with cardiotoxic agents, should have a clinical risk assessment of cardiac function using the New York Heart Association Functional Classification. To be eligible for this trial, patients should be class 2B or better
Ejection fraction \>= 50% (or \>= 45% if no evidence of congestive heart failure \[CHF\] or other cardiac symptoms) by transthoracic echocardiogram (TTE) or multi-gated acquisition (MUGA) scan
White blood cells (WBC) must be \< 25 x 10\^9/L. Hydroxyurea and leukapheresis are permitted to control the WBC prior to enrollment and initiation of protocol-defined therapy but must be stopped within 24 hours of initiation of protocol therapy. Must not have had any signs of leukostasis requiring cytoreduction
Female patients of childbearing potential must agree to use 2 forms of contraception from screening visit until 120 days following the last dose of study treatment. 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 screening visit until 180 days until the last dose of study treatment. They must also refrain from sperm donation from screening visit until 180 days following the last dose of study treatment
Patients must have previously untreated AML with no prior treatment other than hydroxyurea or intrathecal chemotherapy for central nervous system (CNS) prophylaxis/treatment. No chemotherapy for AML outside of hydroxyurea for treatment of leukostasis or all-trans retinoic acid (ATRA) for initially suspected acute promyelocytic leukemia (APL) (that is ruled out) is allowed
Ability to understand and the willingness to sign a written informed consent document. Legally authorized representatives may sign and give informed consent on behalf of study participants

Exclusion

History of allergic reactions attributed to compounds of similar chemical or biologic composition to SNDX-5613, daunorubicin or cytarabine
Patients with uncontrolled intercurrent illness or any other significant condition(s) that would make participation in this protocol unreasonably hazardous
Patient must not have received known strong or moderate CYP3A4 inhibitors, or strong CYP3A4 inducers (with the exception of antifungal prophylaxis with azoles) within 7 days of enrollment. As part of the enrollment/informed consent procedures, the patient will be counseled on the risk of interactions with other agents, and what to do if new medications need to be prescribed or if the patient is considering a new over-the-counter medicine or herbal product
Pregnant women are excluded from this study because SNDX-5613 is a menin-KMT2A inhibitor agent with the potential for teratogenic or abortifacient effects. Because there is an unknown but potential risk for adverse events in nursing infants secondary to treatment of the mother with SNDX-5613, breastfeeding should be discontinued if the mother is treated with SNDX-5613. These potential risks may also apply to other agents used in this study
Isolated myeloid sarcoma (i.e., patients must have blood or marrow involvement with AML to enter the study)
Acute promyelocytic leukemia (French-American-British \[FAB\] M3)
Untreated, active central nervous system (CNS) involvement by AML. Patients are allowed to undergoing diagnostic lumbar puncture (LP) with intrathecal chemotherapy while on study
Uncontrolled symptomatic disseminated intravascular coagulopathy with active bleeding or signs of thrombosis
Patients with Fridericia's correction formula (QTcF) \>= 450 ms at screening; patients with right, left, or partial bundle branch blocks or a pacemaker who are asymptomatic are eligible regardless of QTC if cleared by cardiology for enrollment in the trial. Any factors that increase the risk of QTc prolongation or risk of arrhythmic event such as congenital long QT syndrome or family history of long QT syndrome
Patients who will exceed a lifetime anthracycline exposure of \> 550 mg/m\^2 daunorubicin or equivalent (or \> 400 mg/m\^2 daunorubicin or equivalent in the event of prior mediastinal radiation) if they receive the maximum potential exposure to anthracyclines per protocol (including both induction and reinduction cycles)
Patients with any gastrointestinal issue of the upper gastrointestinal (GI) tract that might affect oral drug absorption or ingestion (eg, gastric bypass, gastroparesis, etc)
Patients who have cirrhosis with a Child-Pugh score of B or C
Patients with Down Syndrome due to higher rates of chemotherapy-associated toxicities, and may have different pharmacokinetics, as well. Toxicities that occur at higher frequencies include cardiotoxicity, a known risk of SNDX-5613 treatment (i.e., QTcF prolongation)
Patients with myelodysplastic syndromes (MDS) treated with previous intensive induction regimens similar to 7+3

What this trial measures

  • Recommended dose for expansion (RDE) for InductionFrom day 1 to 42 of Induction or Re-Induction

    Will be determined based on the totality of safety, tolerability, clinical activity, and pharmacokinetic (PK) data as appropriate. The tolerability of doses administered in cycles after the dose limiting toxicity (DLT) observation window should be taken into account in determination of the RDE. For all patients who receive at least one dose of any of the study drug(s), adverse events will be documented and summarized by type, grade, severity, and attribution using the Common Terminology Criteria for Adverse Events (CTCAE) version (v)5.0 criteria. In addition, the number of treatment cycles received and reasons for going off treatment will be summarized to assess treatment tolerability.

  • RDE for ConsolidationFrom day 1 to 42 of Consolidation or until full count recovery with recovery to grade 1 toxicity from treatment, whichever comes first

    Will be determined based on the totality of safety, tolerability, clinical activity, and PK data as appropriate. The tolerability of doses administered in cycles after the DLT observation window should be taken into account in determination of the RDE.For all patients who receive at least one dose of any of the study drug(s), adverse events will be documented and summarized by type, grade, severity, and attribution using the CTCAE v5.0 criteria. In addition, the number of treatment cycles received and reasons for going off treatment will be summarized to assess treatment tolerability.

  • Recommended phase 2 dose for expansion cohortFrom day 1 of Induction to day 42 of Consolidation or until full count recovery with recovery to grade 1 toxicity from treatment, whichever comes first

    RP2D of induction will be determined based on isotonic regression. Specifically, the RP2D that is selected is the dose for which the isotonic estimate of the toxicity rate is closest to the targeted DLT (i.e., 20%) via "BOIN" software \[MD Anderson\]) and will also factor in data such as PK/pharmacodynamic data to determine the biologically effective dose. For all patients who receive at least one dose of any of the study drug(s), adverse events will be documented and summarized by type, grade, severity, and attribution using the CTCAE v5.0 criteria. In addition, the number of treatment cycles received and reasons for going off treatment will be summarized to assess treatment tolerability.