E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated | |
E.1.1.1 | Medical condition in easily understood language |
acute leukaemia after at least two intensive treatment regimens in paediatric patients
advanced solid tumour in paediatric patients |
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E.1.1.2 | Therapeutic area | Diseases [C] - Cancer [C04] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10048683 |
E.1.2 | Term | Advanced cancer |
E.1.2 | System Organ Class | 100000004864 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 18.0 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10024324 |
E.1.2 | Term | Leukaemias |
E.1.2 | System Organ Class | 10005329 - Blood and lymphatic system disorders |
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E.1.3 | Condition being studied is a rare disease | Yes |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
determination of MTD of volasertib in paediatric patients with cancer |
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E.2.2 | Secondary objectives of the trial |
safety, preliminary efficacy, pharmacokinetics in a paediatric patient population |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal 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
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E.4 | Principal 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 CNS 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
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E.5 End points |
E.5.1 | Primary end point(s) |
1: number of patients with dose limiting toxicity
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
1: number of patients with hepatic injury as defined as protocol-specified adverse events of special interest
2: 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 Eevent grade 3 or higher)
3: number of patients with changes in cardiac activity (prolonged QTc interval) reported as clinically relevant observations ( Adverse Events)
4: number of patients with complete remission (CR)
5: progression free survival
6: overall survival
7: pharmacokinetics
8: number of patients with complete remission with incomplete blood count (CRi)
9: number of patients with partial response/remission (PR)
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1: up to 6 months
2: up to 6 months
3: up to 6 months
4: up to 6 months
5: up to one year
6: up to one year
7: up to six months
8: up to 6 months
9: up to 6 months
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E.6 and E.7 Scope of the trial |
E.6 | Scope of the trial |
E.6.1 | Diagnosis | No |
E.6.2 | Prophylaxis | No |
E.6.3 | Therapy | Yes |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | Yes |
E.6.8 | Bioequivalence | No |
E.6.9 | Dose response | No |
E.6.10 | Pharmacogenetic | No |
E.6.11 | Pharmacogenomic | No |
E.6.12 | Pharmacoeconomic | No |
E.6.13 | Others | No |
E.7 | Trial type and phase |
E.7.1 | Human pharmacology (Phase I) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
Phase I study in paediatric patients |
Phase I |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | No |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | No |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Information not present in EudraCT |
E.8.2.2 | Placebo | Information not present in EudraCT |
E.8.2.3 | Other | Information not present in EudraCT |
E.8.2.4 | Number of treatment arms in the trial | 1 |
E.8.3 |
The trial involves single site in the Member State concerned
| No |
E.8.4 | The trial involves multiple sites in the Member State concerned | Yes |
E.8.4.1 | Number of sites anticipated in Member State concerned | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 11 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | No |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.7 | Trial has a data monitoring committee | Yes |
E.8.8 |
Definition of the end of the trial and justification where it is not the last
visit of the last subject undergoing the trial
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E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 11 |
E.8.9.1 | In the Member State concerned days | 19 |
E.8.9.2 | In all countries concerned by the trial years | 2 |
E.8.9.2 | In all countries concerned by the trial months | 11 |
E.8.9.2 | In all countries concerned by the trial days | 19 |