E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
rhabdomyosarcoma no metastatic |
rabdomiosarcoma no metastásico |
|
E.1.1.1 | Medical condition in easily understood language |
rhabdomyosarcoma no metastatic |
rabdomiosarcoma no metastásico |
|
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 | 14.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10039022 |
E.1.2 | Term | Rhabdomyosarcoma |
E.1.2 | System Organ Class | 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) |
|
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 |
To investigate the role of doxorubicin dose intensity in patients with rhabdomyosarcoma no metastatic included in the High Risk Group |
Investigar el papel de la dosis de doxorubicina en la quimioterapia de intensidad en pacientes con rabdomiosarcoma no metastásico en el Grupo de Alto Riesgo |
|
E.2.2 | Secondary objectives of the trial |
To investigate the role of doxorubicin dose intensity and maintenance chemotherapy in patients with rhabdomyosarcoma no metastatic included in the High Risk Group |
Investigar el papel de la dosis de doxorubicina en la quimioterapia de intensidad y mantenimiento en pacientes con rabdomiosarcoma no metastásico en el Grupo de Alto Riesgo |
|
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
- A pathologically proven diagnosis of Rhabdomyosarcoma. - No evidence of metastatic lesions. - Age less than 21 years (20 years and 364 days) of age. - Previously untreated except for primary surgery. - No pre-existing illness preventing treatment, in particular renal function must be equivalent to grade 0-1 nephrotoxicity, no prior history of cardiac disease and normal shortening fraction (> 28%) and ejection fraction (> 47%). - No previous malignant tumours. - Interval between diagnostic surgery and chemotherapy no longer than 8 weeks. - Diagnostic material available for pathology review. - Available for long term follow up through the treatment centre. - Written informed consent for treatment available. - stratification according to the High Risk Arm - age > 6 months (and < 21 years) - not paratesticular alveolar RMS - informed consent given for the randomised study |
- Pacientes con confirmación patológica de rabdomiosarcoma - Sin evidencia de enfermedad metastásica - Edad 6 m - < 21 años - No tratados previamente excepto por cirugía primaria - Sin enfermedad preexistente que impida tratamiento - Sin tumores malignos previos. - Intérvalo entre cirugía diagnóstica y tratamiento no superior a 8 semanas - Disponibilidad de material diagnóstico para revisión patológica - Disponibilidad de seguimiento a largo plazo a través del centro de tratamiento - Consentimiento informado escrito para disponibilidad de tratamiento - Incluido en el Grupo de Alto Riesgo - Edad > 6 meses (niños menores son elegibles para el protocolo de tratamiento pero no entrarán en el ensayo randomizado) - Consentimiento informado para el estudio randomizado |
|
E.4 | Principal exclusion criteria |
Any negative response mentioned in the "main inclusion criteria" will be grounds for ineligibility of the patient. |
Cualquier respuesta negativa mencionada en el apartado ?principales criterios de inclusión? será motivo de inelegibilidad del paciente. |
|
E.5 End points |
E.5.1 | Primary end point(s) |
Survival, event-free survival, toxicity |
Supervivencia, Supervivencia Libre de Acontecimientos, Toxicidad |
|
E.5.1.1 | Timepoint(s) of evaluation of this end point |
|
E.5.2 | Secondary end point(s) |
Survival, event-free survival, toxicity |
Survival, event-free survival, toxicity |
|
E.5.2.1 | Timepoint(s) of evaluation of this end point |
|
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 | No |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | No |
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 | 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) | No |
E.7.3 | Therapeutic confirmatory (Phase III) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | Yes |
E.8.1.1 | Randomised | Yes |
E.8.1.2 | Open | Yes |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | Yes |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | Yes |
E.8.2.3.1 | Comparator description |
quimioterapia habitual |
usual chemotherapy |
|
E.8.2.4 | Number of treatment arms in the trial | 2 |
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 | 36 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 49 |
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 | 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
|
last visit of the last subject undergoing the trial |
|
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 6 |
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 | 7 |
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 |