E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Enfants de moins de 18 ans, atteints de tumeurs solides et leucémies, traités par la Doxorubicine. |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10047985 |
E.1.2 | Term | Wilms tumor |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10029260 |
E.1.2 | Term | Neuroblastoma |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10041299 |
E.1.2 | Term | Soft tissue sarcomas |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10000844 |
E.1.2 | Term | Acute lymphoblastic leukaemia |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 12.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10015560 |
E.1.2 | Term | Ewing's sarcoma |
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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 |
Détermination de la pharmacocinétique de la doxorubicine chez des enfants atteints de tumeurs solides et leucémies en fonction de l’âge. |
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E.2.2 | Secondary objectives of the trial |
Détermination de la variabilité intra-individuelle, inter-individuelle et résiduelle des paramètres pharmacocinétiques de la doxorubicine chez les enfants. • Détermination de la relation entre les paramètres pharmacocinétiques et les caractéristiques des enfants (age, sexe, poids…) • Evaluation de l’impact pharmacogénétique du polymorphisme des gènes MDR-1, SLC22A16 et CBR sur la clairance de la doxorubicine. • Evaluation du rôle potentiel des peptides natriurétiques et la Troponine comme indicateurs de la cardiotoxicité subclinique. • Corrélation des données pharmacocinétiques avec la toxicité à long terme.
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Patients de moins de 18 ans. • Traités par la doxorubicine pour les tumeurs de Wilms, neuroblastome, sarcome des tissus mous, sarcome d’Ewing ou leucémie lymphoblastique aiguë, selon le protocole national ou européen du traitement de ces tumeurs. • Exceptionnellement, les enfants de moins de 3 ans, traités avec la doxorubicine pour d’autres types de tumeur sont inclus. • Espérance de vie supérieure à trois mois. • Consentement parental ou du représentant légal. • Accord de l’enfant s'il est en âge de comprendre. • Statut de performance de Karnofsky inférieur ou égal à 70%.
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E.4 | Principal exclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
Evaluation de la variation des paramètres pharmacocinétiques et pharmacodynamiques de la doxorubicine chez des enfants de moins de 3 ans et des enfants de 3 à 17 ans. |
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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 | No |
E.6.4 | Safety | No |
E.6.5 | Efficacy | No |
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 | Yes |
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 | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | No |
E.7.1.3.1 | Other trial type description | |
E.7.2 | Therapeutic exploratory (Phase II) | Yes |
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 | 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
| 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 | 6 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 21 |
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 | 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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Le nombre de patients est atteint. Les données de tous les patients sont saisies et validées. La base de donnée est gelée. |
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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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 3 |
E.8.9.2 | In all countries concerned by the trial months | 0 |
E.8.9.2 | In all countries concerned by the trial days | 0 |