Open Dose Escalating Trial to Determine the Maximum Tolerated Dose in Paediatric Patients With Advanced Cancers for Whom no Therapy is Known

Open, Non-controlled, Dose Escalating Phase I Trial to Evaluate the Pharmacokinetics, Pharmacodynamics, Tolerability and Toxicity of Volasertib in Paediatric Patients From 2 Years to Less Than 18 Years of Age With Acute Leukaemia or Advanced Solid Tumour, for Whom no Effective Treatment is Known

The present trial will be performed according to an open design to determine the maximum tolerable dose (MTD) by evaluation of dose-limiting toxicity (DLT) of volasertib in paediatric leukaemia and solid tumours in the age group 2 to less than 12 and 12 to less than 18 years. A further objective is to collect data on safety, tolerability, toxicity, efficacy (preliminary activity), pharmacokinetics and pharmacodynamics of volasertib in paediatric cancer patients

Inclusion criteria:

• paediatric patients with leukaemia or advanced solid tumours including lymphomas (age 2 - less than 18 years) for whom no further treatment is known
• Lansky score > 60 for children 2 to less than 12 years
• Karnofsky score > 60 for children aged 12 or older
• life expectancy of at least 6 weeks as judged by the investigator
• parents or legal guardians have given written informed consent and informed assent suitable for the respective age group obtained

Exclusion criteria:

• patient eligible for other anti-leukaemic therapy with curative intent or effective therapy known for solid tumour therapy
• presence of cardiac disease (LVEF by echocardiography less than 25 %)
• symptomatic Central Nervous System involvement of the malignant disease
• primary CNS tumour
• inadequate lab parameters
• inadequate venous access
• QTc prolongation
• pregnancy, breastfeeding
• other diseases or CTs that might interfere with evaluation of safety