E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
patients in paediatric age (> 6 months & < 21 years old) with localized rhabdomyosarcoma |
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E.1.1.1 | Medical condition in easily understood language |
Rhabdomyosarcoma is a type of cancer (cancer of connective tissues) |
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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 | 16.0 |
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) |
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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 |
To investigate the role of doxorubicin dose intensity and vinorelbine maintenance chemotherapy in patients included in the High Risk Group (randomised trial)
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E.2.2 | Secondary objectives of the trial |
to give an homogeneous local and systemic treatment Europe-wide according to the risk of local and metastatic relapse in patients categorized in Low, Standard and Very High risk Groups (observational study)
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
patients with: a)age > 6 months <21 years; b)pathologically confirmed rhabdomyosarcoma; c) no evidence of metastatic disease; d) previously untreated except for primary surgery.
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E.4 | Principal exclusion criteria |
patients with a)age > 21 years; b)metastatic disease; c)previously treated with CT and/or RT; d) pre-existing illness preventing treatment.
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E.5 End points |
E.5.1 | Primary end point(s) |
Intensification question :
- Primary end point is "event free survival"
Maintenance question
- Primary end point is "disease free survival", measured as time from date of second randomisation up to relapse or death. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
Intensification question :
"Event free survival" measured as time from date of first randomisation up to an event.
Event is defined as: death for all reasons, progression of a residual tumour, relapse following previous complete remission, appearance of a new tumour and switch for a second line chemotherapy in patients without good response.
Maintenance question :
"disease free survival" measured as time from date of second randomisation up to relapse or death. |
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E.5.2 | Secondary end point(s) |
Secondary end points for intensification question are:
• Overall survival
• Progression free survival
• response rate in according to classification criteria reported in chapter 19.
• toxicity according to NCI-CTC version 3
Secondary end points for maintenance question are:
• overall survival
• toxicity according to NCI-CTC version 3 |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
Secondary end points for intensification question are:
• Overall survival, measured as time from date of first randomisation up to death for all reasons.
• Progression free survival, measured as time from date of first randomisation up to tumour progression.
• response rate in according to classification criteria reported in chapter 19.
• toxicity according to NCI-CTC version 3
Secondary end points for maintenance question are:
• overall survival, measured as time from date of second randomisation up to death for all reasons.
• toxicity according to NCI-CTC version 3 |
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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 | 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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
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 | No |
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 | 7 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 145 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Argentina |
Belgium |
Czech Republic |
France |
Ireland |
Israel |
Italy |
Netherlands |
Norway |
Slovakia |
Spain |
Switzerland |
United Kingdom |
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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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For "intensification question", the trial will be terminated after an interim analysis if the main question is answered.
For "maintenance question", the trial will be terminated after the interim analysis if the hypothesis of no treatment difference is rejected. |
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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 | 8 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |
E.8.9.2 | In all countries concerned by the trial years | 8 |