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Carfilzomib (relapsed/refractory ALL)

Clinical study of carfilzomib in children with relapsed or refractory leukemia
Who can enter
  • Children with acute lymphoblastic leukemia (ALL) that has come back (relapse)
    or that did not respond sufficiently to previous treatment (refractory)
  • >1 month till 21 years old


This study has two parts: induction chemotherapy and optional consolidation chemotherapy.

In the induction part of the study, we investigate how safe the drug carfilzomib is in combination with other chemotherapy in infants, children, and young adults with recurrent or refractory acute lymphoblastic leukemia. And how well it works. After the induction therapy the physician will determine, based on the response (effect of the treatment) and possible side effects, whether the child can continue treatment with carfilzomib. The treatment with carfilzomib will then continue during the consolidation chemotherapy treatment.

In the consolidation part of the study, we re-examine how safe the drug carfilzomib is and how well it works. This will look at how well carfilzomib works in combination with chemotherapy.


Carfilzomib was developed by Onyx Inc., a for-profit biopharmaceutical company. Carfilzomib has been approved by Food and Drug Administration (FDA) and regulatory agencies in Europe for use in certain adult patients with relapsed or refractory multiple myeloma. It has not yet been approved as a drug for children with acute lymphoblastic leukemia.

Carfilzomib is a type of drug called a proteasome inhibitor. A proteasome inhibitor works by preventing breakdown of proteins in cells, causing the cells to perish. Cancer cells are more sensitive to these effects than normal cells.

We first treat the child with combination chemotherapy with carfilzomib for 22 days. We call this the induction therapy. If the child tolerates the induction therapy and there is no progression (worsening) of the disease, a second cycle of carfilzomib in combination with the consolidation chemotherapy is possible. The child will then receive 22 days of consolidation chemotherapy with carfilzomib. At any time during treatment, the investigator may recommend stopping treatment with carfilzomib and continuing with another treatment.

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 Office.

Last reviewed

September 14, 2022