NCT05886049RecruitingPhase 1
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
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
Source: ClinicalTrials.gov · Last updated 25 Aug 2026
Brief summary (registry, in English)
This phase Ib trial tests the safety, side effects, and best dose of SNDX-5613 when given in combination with the standard chemotherapy treatment (daunorubicin and cytarabine) in treating patients with newly diagnosed acute myeloid leukemia that has changes in the NPM1 gene or MLL/KMT2A gene. SNDX-5613 blocks signals passed from one molecule to another inside cancer cells that are needed for cancer cell survival. Drugs used in chemotherapy, such as daunorubicin and cytarabine, work in different ways to stop the growth of cancer cells, either by killing the cells, by stopping them from dividing, or by stopping them from spreading. Adding SNDX-5613 to the standard chemotherapy treatment may be able to shrink or stabilize the cancer for longer than the standard chemotherapy treatment alone.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1
- Study type
- Interventional
- Enrollment
- 38 (estimated)
- First submitted
- 1 Jun 2023
- Start date
- 20 Jun 2024
- Primary completion date
- 31 Dec 2027
- Lead sponsor
- National Cancer Institute (NCI)
- Diseases
- Acute myeloid leukemia
- NCT number
- NCT05886049
What you commit to
- Study type: Interventional · Phase 1
- Planned participants: 38
- From 20 Jun 2024 to the primary completion date, 31 Dec 2027
- 23 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
- Untreated, active central nervous system (CNS) involvement by AML. Patients are allowed to undergoing diagnostic lumbar puncture (LP) with intrathecal chemotherapy while on study
- 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 with myelodysplastic syndromes (MDS) treated with previous intensive induction regimens similar to 7+3
Other illness
- 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
- Uncontrolled symptomatic disseminated intravascular coagulopathy with active bleeding or signs of thrombosis
- 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)
Lab values
- 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
Other
- 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)
- Patients who have cirrhosis with a Child-Pugh score of B or C
Who the study is looking for
- 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
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (23)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| UM Sylvester Comprehensive Cancer Center at Aventura Coordinator of this site? Confirm its status | Aventura | United States | Recruiting |
| University of Maryland/Greenebaum Cancer Center Coordinator of this site? Confirm its status | Baltimore | United States | Recruiting |
| Dana-Farber Cancer Institute Coordinator of this site? Confirm its status | Boston | United States | Recruiting |
| UNC Lineberger Comprehensive Cancer Center Coordinator of this site? Confirm its status | Chapel Hill | United States | Recruiting |
| Carolinas Medical Center/Levine Cancer Institute Coordinator of this site? Confirm its status | Charlotte | United States | Recruiting |
| University of Virginia Cancer Center Coordinator of this site? Confirm its status | Charlottesville | United States | Active, not recruiting |
| University of Chicago Comprehensive Cancer Center Coordinator of this site? Confirm its status | Chicago | United States | Recruiting |
| University of Cincinnati Cancer Center-UC Medical Center Coordinator of this site? Confirm its status | Cincinnati | United States | Recruiting |
| Ohio State University Comprehensive Cancer Center Coordinator of this site? Confirm its status | Columbus | United States | Recruiting |
| UM Sylvester Comprehensive Cancer Center at Coral Gables Coordinator of this site? Confirm its status | Coral Gables | United States | Recruiting |
| UM Sylvester Comprehensive Cancer Center at Deerfield Beach Coordinator of this site? Confirm its status | Deerfield Beach | United States | Recruiting |
| University of Kansas Clinical Research Center Coordinator of this site? Confirm its status | Fairway | United States | Recruiting |
| University of Kansas Cancer Center Coordinator of this site? Confirm its status | Kansas City | United States | Recruiting |
| University of Miami Miller School of Medicine-Sylvester Cancer Center Coordinator of this site? Confirm its status | Miami | United States | Recruiting |
| University of Oklahoma Health Sciences Center Coordinator of this site? Confirm its status | Oklahoma City | United States | Recruiting |
| UC Irvine Health- Chao Family Comprehensive Cancer Center Coordinator of this site? Confirm its status | Orange | United States | Recruiting |
| University of Kansas Hospital-Indian Creek Campus Coordinator of this site? Confirm its status | Overland Park | United States | Recruiting |
| UM Sylvester Comprehensive Cancer Center at Plantation Coordinator of this site? Confirm its status | Plantation | United States | Recruiting |
| University of California Davis Comprehensive Cancer Center Coordinator of this site? Confirm its status | Sacramento | United States | Suspended |
| Huntsman Cancer Institute/University of Utah Coordinator of this site? Confirm its status | Salt Lake City | United States | Recruiting |
| University of Cincinnati Cancer Center-West Chester Coordinator of this site? Confirm its status | West Chester | United States | Recruiting |
| University of Kansas Hospital-Westwood Cancer Center Coordinator of this site? Confirm its status | Westwood | United States | Recruiting |
| Wake Forest University Health Sciences Coordinator of this site? Confirm its status | Winston-Salem | United States | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.