A Phase 1 Study of Ziftomenib Plus Chemotherapy in Children and Young Adults With Acute Leukemia

Full Title

A Phase 1 Trial of the Menin Inhibitor Ziftomenib in Combination with Chemotherapy for Children with Relapsed/Refractory KMT2A-rearranged, NUP98-rearranged, or NPM1-mutant Acute Leukemia

Purpose

Researchers are doing this study to find the best dose of ziftomenib to give with standard chemotherapy in children and young adults with acute leukemia. The people in this study have acute leukemia that keeps growing or came back after treatment. In addition, their cancers have a mutation (change) in the KMT2A, NUP98, or NPM1 genes.

Participants in this study will have:

• Acute lymphoblastic leukemia (ALL)
• Acute undifferentiated leukemia (AUL)
• Mixed phenotype acute leukemia (MPAL), or
• Acute myeloid leukemia (AML)

Ziftomenib works by blocking menin, a protein that plays a role in cancer cell growth. By blocking menin, your cancer may stop growing and go into remission. It is taken orally (by mouth).

If you or your child takes part in this study, you will take ziftomenib and also get fludarabine and cytarabine. These are standard chemotherapy drugs.

Who Can Join

To join this study, there are a few conditions. You or your child must:

• Have ALL, AUL, MPAL, or AML that keeps growing or came back after treatment.
• Have cancer with a mutation in the KMT2A, NUP98, or NPM1 genes.
• Be age 21 years or younger.

Contact

For more information or to see if you or your child can join this study, please call 833-MSK-KIDS.