E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Advanced and pre-treated Ewing’s Sarcoma |
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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 | LLT |
E.1.2 | Classification code | 10015562 |
E.1.2 | Term | Ewing's sarcoma metastatic |
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E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9.1 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10015564 |
E.1.2 | Term | Ewing's sarcoma recurrent |
|
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 | 10015562 |
E.1.2 | Term | Ewing's sarcoma metastatic |
|
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 | 10015564 |
E.1.2 | Term | Ewing's sarcoma recurrent |
|
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 main objective of this trial is to determine the effectiveness of oral treosulfan in patients with advanced pre-treated Ewing's Sarcoma. |
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E.2.2 | Secondary objectives of the trial |
The secondary objectives include: 1. Event-free survival 2. Overall survival 3. Toxicity 4. Duration of response 5. Time to progression/relapse 6. Assessment of the correlation between the formation and repair of treosulfan-induced DNA interstrand crosslinks (ICL) on response and toxicity
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Age 3 - 50 years 2. Histologically proven Ewing’s sarcoma/Peripheral Neuroectodermal Tumour 3. Patients with advanced, refractory disease who have failed at least one course of conventional chemotherapy and for whom no curative option exists. 4 .Measurable disease, defined by RECIST 5. Evidence of disease progression within the preceding 8 weeks 6. Adequate blood counts and biochemical profile 7. WHO Performance status 0-2 (patients > =16 years); or Lansky Performance Status > =30. (n.b. patients with WHO performance status 3 due to spinal disease will be eligible provided they are otherwise medically well) 8. Patient able to comply with protocol treatment (swallow capsules) and follow up 9. Life expectancy of 3 months or greater 10. Written informed consent of patient or parent/legal guardian. |
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E.4 | Principal exclusion criteria |
1. Newly diagnosed, or resectable Ewing’s sarcoma 2. Pregnant/lactating women, or women of child bearing potential unless using effective contraception 3. Concurrent treatment with any other anti-cancer therapy, except palliative radiotherapy to non-target lesions 4. Concurrent treatment with other experimental drugs 5. Concurrent treatment with growth factors |
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E.5 End points |
E.5.1 | Primary end point(s) |
Primary endpoint: Objective response rate achieved using RECIST |
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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 | Yes |
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 | 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) | 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 | No |
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 | 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
| |
E.8.9 Initial estimate of the duration of the trial |
E.8.9.1 | In the Member State concerned years | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |