E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
To measure concentrations of liposomal cytarabine in CSF and plasma over time after intrathecal administration of liposomal cytarabine in children of different age groups with a malignant brain tumor and leptomenigeal dissemination. Measurements of concentrations of cytarabine after intrathecal administration of liposomal cytarabine will help to ensure proper dosage of liposomal cytarabine in small children, avoiding neurotoxic events and subtherapeutic drug levels. |
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MedDRA Classification |
E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
CSF: AUC, t1/2, Cmax, Cmin, Cav(ss) |
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E.2.2 | Secondary objectives of the trial |
Frequency and degree of toxic reactions, adverse events, and leukencephalopathy; plasma: AUC, t1/2, Cmax, Cmin, Cav(ss) |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
• Female or male, aged between 0 and 19 years (at time of first study dose) • Malignant brain tumor with leptomeningeal dissemination or risk of leptomeningeal dissemination, for whom no routine protocol is available (for example, AT/RT or Infantile malignant glioma) OR: Relapse of a brain tumor with leptomeningeal dissemination or risk of leptomeningeal dissemination of any histology • Treatment with intrathecal administered liposomal cytarabine for therapeutic reasons • Life expectancy of at least 8 weeks • Written informed consent of parents • Serum creatinine < 1.5 mg/L • Total serum bilirubin < 2.0 mg/dL and ALT less than 5 times upper limit of normal • Platelet count > 40.000/mm3 within 48 hours before first treatment
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E.4 | Principal exclusion criteria |
• Treatment with liposomal cytarabine not indicated • Evidence of obstructive hydrocephalus or compartmentalization of CSF flow • Severe uncontrolled infection
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E.5 End points |
E.5.1 | Primary end point(s) |
Pharmacokinetics of liposomal cytarabine in CSF of infants with brain malignancy |
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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 | No |
E.6.6 | Pharmacokinetic | Yes |
E.6.7 | Pharmacodynamic | No |
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) | No |
E.7.1.1 | First administration to humans | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
E.7.3 | Therapeutic confirmatory (Phase III) | Information not present in EudraCT |
E.7.4 | Therapeutic use (Phase IV) | Information not present in EudraCT |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
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.8.3 |
The trial involves single site in the Member State concerned
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.5 | The trial involves multiple Member States | No |
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 | Information not present in EudraCT |
E.8.7 | Trial has a data monitoring committee | No |
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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enrollment of 15 patients |
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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 | 4 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |