AUGMENT-102: A Phase 1 Study of SNDX-5613 with Chemotherapy for Patients with Leukemias That Have Certain Genetic Changes


Full Title

AUGMENT-102: A Phase 1, Open-label, Dose-escalation Study to Evaluate Safety, Tolerability and Preliminary Anti-Leukemic Activity of SNDX-5613 in Combination with Chemotherapy in Patients with Relapsed/Refractory Leukemias Harboring Alterations in KMT2A/MLL, Nucleophosmin 1 (NPM1), and Nucleoporin 98 (NUP98) Genes


The purpose of this study is to assess the safety and effectiveness of the investigational drug SNDX-5613 when given with standard chemotherapy in children and adults with fast-growing leukemia that has come back or continued to grow despite treatment and contains certain genetic mutations. SNDX-5613 is designed to block a protein called menin, which is required for cells to divide. By blocking this protein, SNDX-5613 may prevent cancer cells from growing. SNDX-5613 is taken orally (by mouth) and can also be given through a nasogastric or g-tube (a tube inserted down the nose into the stomach).

Who Can Join

To be eligible for this study, patients must meet several requirements, including:

  • Participants must have fast-growing leukemia that has come back or continued to grow despite prior treatment.
  • Patients’ leukemia must contain a mutation in the KMT2A/MLL and/or the NPM1 gene.
  • Patients must be able to walk and do routine activities for more than half of their normal waking hours.
  • This study is for people age 30 days and older.


For more information about this study and to inquire about eligibility, please contact 1-833-MSK-KIDS.




Phase I (phase 1)

Disease Status

Relapsed or Refractory


Co-Investigators ID