E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
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 a novel combination of mIBG given at a higher dose than usual in conjunction with topotecan. There is good experimental evidence to indicate that this may yield better results. Primary resistant high risk neuroblastoma: Assess a salvage schedule of treatment for patients with high-risk neuroblastoma treated with rapid COJEC in the SIOP-Europe High-Risk Study whose disease has failed to respond sufficiently to proceed to surgery for the primary tumour.
Relapsed stage 4 neuroblastoma: Assess a palliative schedule of treatment for patients with stage 4 neuroblastoma who have relapsed after intensive treatment including high dose chemotherapy with haemopoietic progenitor cell support.
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E.2.2 | Secondary objectives of the trial |
Secondly, this study aims to investigate, where possible, the radiation dose received by the cancer (as distinct from the activity of radiation administered to the patient), and also the value of PET scanning in addition to mIBG scanning in the assessment of disease extent and the response to treatment. |
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E.2.3 | Trial contains a sub-study | Information not present in EudraCT |
E.3 | Principal inclusion criteria |
1) Is aged 1 year or over and has high-risk neuroblastoma (INSS Stage 4, or Stage 2 or 3 with MycN amplification) 2) Is enrolled on the SIOP-Europe high-risk neuroblastoma study or has been treated according to that protocol, or has been treated according to a similar protocol 3) Fails to achieve satisfactory remission with induction chemotherapy (defined as >50% reduction or more than 3 positive sites on mIBG scintigraphy, or persistent cytomorphological positive disease in bone marrow aspirates or trephine biopsies) at day 80 after COJEC induction, (or progressive disease at an earlier date necessitating a change to treatment) 4) has mIBG positive disease on diagnostic scintigraphy at the time of consideration of entry to this study 5) is being treated at, or clinicians and parents (or the patient in the case of an adult) are willing for the patient to be referred to, a centre participating in this study 6) has parents who are willing to consider entry into this study and who give informed consent in writing or, in the case of a competent adult, the patient may give informed consent on their own.
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E.4 | Principal exclusion criteria |
Not meeting any of the inclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary resistant high risk neuroblastoma: 1. The proportion of patients who respond to treatment (PR and CR at metastatic sites), evaluated by mIBG scintigraphy in conjunction with, where possible, PET and CT imaging. 2. The proportion of patients who, as a result of this treatment, are able to progress to potentially curative treatment with surgery, and further systemic treatment. 3. To perform, where possible, tumour dosimetry to see whether the tumour absorbed radiation dose (as distinct from the whole body measurement) correlates with response in that tumour.
Relapsed stage 4 neuroblastoma: 1. The proportion of patients who respond to treatment (PR and CR at metastatic sites), evaluated by mIBG scintigraphy in conjunction with, where possible, PET and CT imaging. 2. The time to tumour progression. 3. To perform, where possible, tumour dosimetry to see whether the tumour absorbed radiation dose (as distinct from the whole body measurement) correlates with response in that tumour.
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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 | Information not present in EudraCT |
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 | No |
E.8.1.2 | Open | No |
E.8.1.3 | Single blind | No |
E.8.1.4 | Double blind | No |
E.8.1.5 | Parallel group | No |
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) | No |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
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.8.5 | The trial involves multiple Member States | Yes |
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 | Information not present in EudraCT |
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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Accrual of target numbers of 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 | 3 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | |
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 |