E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Paediatric cancer patients (aged 2 to < 18 years) with Ewing’s sarcoma/soft tissue sarcoma, neuroblastoma, brain tumours, and all other malignancies (excluding leukaemia) who are planned to undergo high dose chemotherapy followed by autologous haematopoietic stem cell transplantation (HSCT) rescue |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10041299 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10029260 |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | HLGT |
E.1.2 | Classification code | 10029211 |
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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 |
The primary objective of this study is to confirm the appropriate dose and efficacy, and to characterise the safety, pharmacokinetics and pharmacodynamics of plerixafor across age and size in paediatric cancer patients when given in addition to standard mobilisation of HSCs into peripheral blood. E. |
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E.2.2 | Secondary objectives of the trial |
The secondary objective of this study is to confirm the efficacy and safety of plerixafor in addition to standard mobilisation of HSCs into peripheral blood, and subsequent collection by apheresis, in paediatric cancer patients. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Age 2 to <18 years 2. Ewing’s sarcoma, soft tissue sarcoma, neuroblastoma, brain tumours or other malignancy (excluding any form of leukaemia) requiring treatment with high dose chemotherapy and autologous transplant as rescue therapy 3. Eligible for autologous transplantation 4. Recovered from all acute significant toxic effects of prior chemotherapy 5. Adequate performance status – for patients >= 16 years of age, defined as Karnofsky score >60 – for patients <16 years of age, defined as Lansky score >60 6. Absolute neutrophil count >1.0 � 10P9/L 7. Platelet count >75 � 10P9/L 8. Calculated creatinine clearance (using the Schwartz method): – during study Stage 1, >80 mL/min/1.73mP2 – during study Stage 2, >60 mL/min/1.73mP2 9. Aspartate aminotransferase/serum glutamic oxaloacetic transaminase (AST/SGOT), alanine aminotransferase/serum glutamic pyruvic transaminase (ALT/SGPT) and total bilirubin <3 � upper limit of normal 10. The patient and/or their parent/legal guardian is willing and able to provide signed informed consent |
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E.4 | Principal exclusion criteria |
1. Any form of leukaemia 2. A co-morbid condition which, in the view of the Investigator, renders the patient at high-risk from treatment complications 3. Previous stem cell transplantation 4. Patients with tumours frequently involving bone marrow (e.g., lymphomas and neuroblastoma) will be expected to have had evaluation of their marrow as part of their standard staging evaluations. Persistent high percentage marrow involvement prior to mobilisation will be prohibited. (Specific guidelines for different indications are provided in Section 9.2.1 with details of bone marrow examination requirements at Screening) 5. A residual acute medical condition resulting from prior chemotherapy 6. Acute infection 7. Fever (temperature >38.5�C) - if fever is between 37�C and 38.5�C, infection must be excluded as a cause 8. Pulse oximetry ≤92% 9. Known HIV positive 10. Positive pregnancy test in post pubertal girls 11. History of clinically significant cardiac abnormality or arrhythmia 12. Use of an investigational drug which is not approved in any indication either in adults or paediatrics within 4 weeks prior to the first dose of G-CSF to be administered as part of the patient’s planned standard mobilisation regimen, and/or during the study up until engraftment of the transplant. Drugs approved for other indications that are being used in a manner considered standard of care for this transplant procedure are allowed 13. The patient (and/or their parent/legal guardian), in the opinion of the Investigator, is unable to adhere to the requirements of the study |
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary efficacy endpoint will be the difference between the 2 treatment arms in Stage 2 (comparative part of the study) in the proportion of patients achieving at least a doubling of peripheral blood CD34+ count from the morning of the day preceding the first apheresis day to the morning prior to apheresis. |
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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 | Yes |
E.6.5 | Efficacy | Yes |
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 | 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) | Yes |
E.7.1.1 | First administration to humans | No |
E.7.1.2 | Bioequivalence study | No |
E.7.1.3 | Other | Yes |
E.7.1.3.1 | Other trial type description |
studio pediatrico dove nella fase I dello studio si valuta il diverso profilo farmacocinetico rispet |
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E.7.2 | Therapeutic exploratory (Phase II) | No |
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 | 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 | Yes |
E.8.1.7.1 | Other trial design description |
fase 2 solo: studio comparativo randomizzato |
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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 | Yes |
E.8.2.3.1 | Comparator description |
fase 2 solo: confronto con mobilizzazione standard |
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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 | 4 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 15 |
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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Si faccia riferimento al protocollo |
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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 | 6 |
E.8.9.1 | In the Member State concerned months | 8 |
E.8.9.1 | In the Member State concerned days | 0 |
E.8.9.2 | In all countries concerned by the trial years | 6 |
E.8.9.2 | In all countries concerned by the trial months | 8 |
E.8.9.2 | In all countries concerned by the trial days | 0 |