Who can enter
- Children with acute myeloid leukemia (AML) in whom the disease has returned (relapsed) or responded insufficiently to treatment (refractory)
- Age: 1 up to and including 21 years old
The goal of this study is to find out how safe the new drug Vyxeos® is, in combination with clofarabine, when given to children and young adults with relapsed/refractory acute myeloid leukemia. This study will establish a safe dosage of Clofarabine in combination with Vyxeos®.
Vyxeos® is a new drug that combines two known drugs (cytarabine and daunorubicin) together in a fixed ratio. This is enclosed in a lipid sphere (liposome), allowing the drugs to access leukemia cells more efficiently.
A study of 309 adults with AML showed that Vyxeos® significantly improved the average duration of survival. Preliminary results from this study are promising. Of the 38 children with relapsed/refractory AML who have received Vyxeos® in study settings to date, approximately 80% respond well to treatment.
In this study, clofarabine is added to treatment with Vyxeos®, with the goal of further improving the efficacy of treatment. Clofarabine is usually administered in combination with other chemotherapy.
In a study in 34 children with relapsed/refractory AML treated with a combination of clofarabine, cytarabine and liposomal daunorubicin, 68% of patients responded to treatment. The side effects were mainly infections. After treatment, 22 patients were able to proceed to stem cell transplantation.