HEM-iSMART subprotocol C (ALL/LBL)
Clinical study of ruxolitinib and venetoclax for children with relapsed or refractory leukemia or lymphoma with mutations in the IL-7R/JAK-STAT signalling pathway.
Recruiting
Who can enter
Children with acute lymphoblastic leukemia (ALL) or lymphoblastic lymphoma (LBL) with mutations in the IL-7R/JAK-STAT signaling pathway, who have had an inadequate response to previous treatment (refractory) or whose disease has come back (relapse)
Age: 1 to 21 years
Goal
Goal
The goal of this study is to investigate whether the combination of the drugs ruxolitinib and venetoclax is tolerable, safe and effective in combination with dexamethasone, cyclophosphamide and cytarabine. This is tested in patients who have had an inadequate response to previous treatments (refractory) or whose disease has come back (relapse).
Background
Background
Certain changes (mutations) present in leukemia or lymphoma cells are responsible for increased and uncontrolled cell growth. Known changes that act like this are mutations in several genes that are part of the IL7R/JAK-STAT signaling pathway. Certain proteins control the growth of many cancers. Amongst those are the so-called tyrosine kinases (such as JAK and STAT kinase).
The drug ruxolitinib inhibits the action of these enzymes, thereby inhibiting cell divisions and thus the growth of leukemia or lymphoma cells. Venetoclax targets a particular protein that is increased in leukemia or lymphoma cells and prevents the cancerous cells from dying naturally (this protein is called: BCL2 protein). Venetoclax inhibits this BCL2 protein, enabling the leukemia cells to die.
Dexamethasone, cyclophosphamide and cytarabine are drugs that can cause leukemia and lymphoma cells to die using other mechanisms of action.
In order to participate in this study please contact your/your child’s treating physician.
For international patients: please feel welcome to contact our International Patients Office.
Last reviewed
Last reviewed
October 30, 2025
Study details
- Study details
Official title
International proof of concept therapeutic stratification trial of molecular anomalies in relapsed or refractory hematological malignancies in children – Sub-Protocol C: Ruxolitinib + venetoclax + dexamethasone, cyclophosphamide and cytarabine in pediatric patients with relapsed or refractory hematological malignanciesCancer type
Acute lymphoblastic leukemia (ALL)
Lymphoblastic lymphoma (LBL)
Phase
1/2Maximum number of patients
26, of whom 3 are expected to participate in the NetherlandsStart date
October 30, 2025Status
OpenLocal principal investigator
Dr. Britta VormoorSponsor
Princess Máxima Center for pediatric oncologyApproval
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 Clinical Trials: 2022-501867-42-01
ClinialTrials.gov: NCT05745714
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.