Who can enter
- 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.
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.
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.