SNDX-5613-0700 (relapsed/refractory leukemias)

Clinical study of SNDX-5613 in patients with leukemias.

Active, not recruiting / Results published

Who can enter

Children with:

Acute Lymphoblastic Leukemia (ALL) harboring a 'mixed lineage leukemia (MLL)' gene rearrangement
Acute Myeloid Leukemia (AML) harboring a 'mixed lineage leukemia (MLL)' gene rearrangement
Acute Myeloid Leukemia (AML) with a nucleophosmin 1 (NPM1)-mutation
Patients (0-18 years) weighing at least 40kg or more may be included

Goal

The goal of this study is to determine how safe the new drug SNDX 5613 is in the treatment of relapsed or refractory ALL, and to find out what the possible side effects might be in children.

Background

We are conducting this clinical study to find out more about blood cancer.

This is the first study of SNDX-5613 in humans. It is a medicine taken by mouth that disturbs a protein in your body called menin. We have already conducted research on this medicine in animals. This showed that animals with leukemia who received SNDX-5613 lived longer than animals who did not. Therefore we want to find out how the medicine works in humans.

The study consists of two phases. In the first phase, we will establish the safe dose of the medicine SNDX-5613. In phase 2, that established safe dose will be used to see how well the medicine works, and additional safety information will be collected. The Netherlands will only participate in the phase 2 part of the study.

Phase 2, the 'dose extension', therefore only starts after the safe dose of the study drug has been determined in phase 1.

This study is now closed for inclusion.

Last reviewed

March 23, 2026

Study details
Official title
A Phase 1/2, Open-label, Dose-Escalation and Dose-Expansion Cohort Study of SNDX-5613 in Patients with Relapsed/Refractory Leukemias, Including Those Harboring an MLL/KMT2A Gene Rearrangement or Nucleophosmin 1 (NPM1) Mutation
Cancer type
- Acute Lymphoblastic Leukemia (ALL) harboring a 'mixed lineage leukemia (MLL/KMT2A)' gene rearrangement
- Acute Myeloid Leukemia (ALL) harboring a 'mixed lineage leukemia (MLL/KMT2A)' gene rearrangement
- Acute Myeloid Leukemia (AML) with a nucleophosmin 1 (NPM1)-mutation
Phase
1/2
Maximum number of patients
222, of whom 6 are expected to participate in the Netherlands
Start date
May 2022
Status
Closed for inclusion
Local principal investigator
Prof. dr. C.M. Zwaan
Sponsor
Syndax Pharmaceuticals, Inc
Approval
The study of this new treatment has been reviewed by an accredited medical research ethics committee. This committee has decided that it is justified to ask patients to participate in this study. More information can be found at: CCMO.
Trial registry number
ClinialTrials.gov: NCT04065399

The above information is intended as a brief summary only and may not reflect the most up-to-date information. For full details and the current status of a protocol, physicians can contact the Princess Máxima Center directly.

Published results

Summary

Aggressive leukemia responds to new drug revumenib

A new group of drugs is effective in treating certain types of leukemia. These drugs are used to treat children and adults with a rare genetic abnormality called KMT2A rearrangement. This abnormality occurs in approximately 1 in 10 people with acute leukemia. The disease is often aggressive and difficult to treat.

Researchers have investigated whether the drug revumenib works in children and adults whose leukemia has returned or did not respond well to standard treatment. Revumenib is a menin (protein) inhibitor. The drug switches off the genetic KMT2A abnormality that forms a protein complex together with menin. Because the drug blocks the formation of this protein complex, some leukemia cells disappear.

The study shows that nearly 1 in 4 children (23%) went into remission after treatment with revumenib. This means that no leukemia cells were found in their blood or bone marrow. This is a clear improvement over previous treatments, when 1 in 10 children went into remission.

A total of 94 people from different countries participated in this study, including 23 children. Five children were treated at the Princess Máxima Center. The Máxima was the only European hospital to participate in this study.

Children responded just as well to the treatment as adults. They were given an adjusted dose of the medication. Nearly 65% of all participants showed a positive response to the medication. In these cases, the leukemia disappeared completely or partially.

New type of medicine

Revumenib belongs to a new group of medicines: menin inhibitors. These medicines block a protein (menin). In children and adults with a KMT2A abnormality, this protein causes leukemia cells to grow. By inhibiting this protein, the leukemia cells can disappear.

The drug also has advantages in terms of quality of life. It is administered as a drink or pill and does not usually cause nausea. In the future, it may even be administered on an outpatient basis, so your child will not have to stay in the hospital.

Next step: new study

The Princess Máxima Center will soon start a new study with another drug from this group: ziftomenib. This drug is also a menin inhibitor. Researchers want to determine the safe dosage for children with acute leukemia and a specific genetic abnormality. The study is being led by Prof. Michel Zwaan's research group and coordinated by the Máxima's Trial and Data Center, together with the American Leukemia Lymphoma Society.

The ultimate goal is to add these drugs to the standard treatment. We hope that this will enable more children with an aggressive form of leukemia to recover in the future.

Would you like to read the scientific publication? Please look here: Issa GC, et al. Menin Inhibition With Revumenib for KMT2A-Rearranged Relapsed or Refractory Acute Leukemia (AUGMENT-101). J Clin Oncol. 2025 Jan;43(1):75-84.