Ziftoped (acute leukemia)
Clinical study of ziftomenib in combination with chemotherapy for children with relapsed/refractory acute leukemia.
Recruiting
Who can enter
Children, adolescents and young adults with recurrent (relapsed) or resistant (refractory) acute leukemia with an abnormality in the KMT2A, NUP98 or NPM1 gene
Age: 0 to 21 years of age
Goal
Goal
In this study, we will evaluate how safe the new drug ziftomenib is in combination with chemotherapy in children, adolescents and young adults. We also want to know how well it works. We will test ziftomenib in different doses in patients with acute leukemia in whom the disease has recurred or in whom the disease has not responded sufficiently to standard treatment.
We will also study how much ziftomenib enters the blood and how quickly it is cleared from the body. This is called pharmacokinetics (PK). We do this by measuring the amount of ziftomenib in the blood.
Background
Background
Children, adolescents and young adults with recurrent or resistant acute leukemia with a gene abnormality in KMT2A, NUP98 or NMP1 have limited treatment options. New drugs, called menin inhibitors, are in development. These can specifically inhibit the cell growth of leukemia cells with an abnormality in the KMT2A, NUP98 or NPM1 gene.
Ziftomenib is one such menin inhibitor. This new drug has already been studied in adults. It was very well tolerated and appeared to be effective. Aside from this drug, adults received no other therapy, such as chemotherapy.
In this clinical trial, we want to study ziftomenib in children, adolescents and young adults up to 21 years of age. We think that if we combine ziftomenib with chemotherapy, it will be even more effective.
When participating, the study lasts up to 1.5 years, with a monitoring period of about 2 years thereafter. Ziftomenib is taken daily; this can be done at home. For the study, at a number of times additional tests and examinations will be done that are different from normal care. A few times, a little more blood or bone marrow is taken; this does not require participants to be pricked extra. Also, DNA testing is done to see what DNA abnormalities are present in the leukemia. Participants will be asked to keep a diary about medication intake. And they will be asked to complete a questionnaire three times about the taste and texture of ziftomenib.
In order to participate in a study please refer to your/your child’s doctor.
For international patients: please feel welcome to contact our International Patients Office.
Last reviewed
Last reviewed
March 23, 2026
Study details
- Study details
Official 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 LeukemiaCancer type
Acute myeloid leukemia
Mixed phenotype leukemia
Acute lymphoblastic leukemia
Phase
1Maximum number of participants
20, of whom 4 are expected to participate in the NetherlandsStart date
March 27, 2025Status
OpenLocal principal investigator
Prof. dr. C.M. ZwaanSponsor
Prinses Máxima Centrum voor kinderoncologieApproval
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
EU CT number: 2023-505262-28-00
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.