A Study to Find a Safe Dose of Volasertib Given in Addition to Standard Salvage Chemotherapy in Children (Age 3 Months to Less Than 18 Years) With Acute Myeloid Leukaemia, in Whom Front-line Chemotherapy Failed
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This is an open-label, dose-escalating trial to evaluate the MTD and/or dose to be used for further development by evaluation of DLT in course 1 and the safety of volasertib when added to standard intensive salvage chemotherapy with DNX-FLA in paediatric patients with AML after failure of first-line therapy. Furthermore, data on efficacy and PK/PD of volasertib in paediatric patients with AML when added to standard intensive salvage chemotherapy will be collected.
Open-label, Dose-escalating Trial to Evaluate the Tolerability, Toxicity, Safety, Pharmacokinetics, Pharmacodynamics and Activity of Volasertib Added to the Standard Intensive Salvage Chemotherapy Regimen With Liposomal Daunorubicine, Fludarabine and Cytarabine (DNX-FLA) Followed by Fludarabine and Cytarabine (FLA) in Children From 3 Months to Less Than 18 Years of Age With Acute Myeloid Leukaemia After Failure of the Front-line Therapy
Study Start Date :
Estimated Primary Completion Date :
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Determination of the maximal tolerated dose of volasertib or the recommended volasertib dose for further studies in combination with standard salvage therapy in paediatric patients with AML after failure of the front-line intensive chemotherapy regimen [ Time Frame: 4 weeks ]
Secondary Outcome Measures :
Anti-leukaemic activity of volasertib in combination with standard salvage therapy [ Time Frame: 8 weeks ]
Event-free survival (EFS) [ Time Frame: up to 5 years ]
Overall survival (OS) [ Time Frame: up to 5 years ]
Number of patients with clinically relevant lab value changes of calcium (hyper- and/or hypocalcaemia) as judged by the investigator and reported as adverse events (Common Terminology Criteria for Adverse Events (CTCAE) grade 3 or higher) [ Time Frame: 8 weeks ]
Number of patients with changes in cardiac activity (prolonged QTc interval) reported as clinically relevant observations (i.e. Adverse Events) [ Time Frame: 8 weeks ]
Predose concentration of volasertib before administration of second dose [ Time Frame: 8 weeks ]
Area under the concentration-time curve of volasertib [ Time Frame: 8 weeks ]
Terminal half-life of volasertib in plasma [ Time Frame: 8 weeks ]
Maximum concentration of volasertib [ Time Frame: 8 weeks ]
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Layout table for eligibility information
Ages Eligible for Study:
3 Months to 17 Years (Child)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Patients 3 months to <18 years of age at the time of informed consent
Patients with AML after failure of the front-line intensive AML therapy
Lansky score at screening >=50 for patients from 3 months to <12 years
Karnofsky score at screening >=50 for patients from 12 to <18 years
Use of highly effective methods of birth-control, if sexually active
Parents/legal guardians and patients have given written informed consent and informed assent suitable for the respective age group
Acute promyelocytic leukaemia and treatment-related AML