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
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
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.