E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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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 | 10029260 |
E.1.2 | Term | Neuroblastoma |
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E.1.3 | Condition being studied is a rare disease | No |
E.2 Objective of the trial |
E.2.1 | Main objective of the trial |
The study is designed as a pilot study to: •Investigate the feasibility of implementing 13-cis-Retinoic acid dose modification following course 1 of treatment, based on targeted 13-cis-Retinoic acid plasma concentrations and observed toxicity.
• Minimize the large inter-patient variation in plasma concentrations of 13-cis-Retinoic acid observed following standard treatment for high-risk neuroblastoma. This will ensure that patients are not exposed to potentially sub-optimal plasma concentrations of 13-cis-RA during long-term treatment, particularly for those children who are not able to swallow 13-cis-RA capsules.
• Obtain preliminary data investigating the potential impact of 13-cis-RA therapeutic monitoring on clinical response and toxicity in children with high-risk neuroblastoma |
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E.2.2 | Secondary objectives of the trial |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
a) Age less than or equal to 18 years. b) Diagnosis of high-risk neuroblastoma. c) Receiving 13-cis-RA (Roaccutane) as part of clinical treatment. d) Single or double lumen central venous catheter in place. e) Written informed consent. f) Protocol approval by national and local ethics committee, regulatory authority and Trust R&D departments. g) A negative pregnancy test for women of child bearing potential, and sexually active patients and partners agreeing to undertake adequate contraceptive measures |
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E.4 | Principal exclusion criteria |
Failure to comply with any of the inclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
Target accrual of up to 75 patients reached
Effective individualised dosing of isotretinoin in high-risk neuroblastoma patients to achieve consistent plasma 13-cis-retinoic acid concentrations >2µM with acceptable toxicity |
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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 | Information not present in EudraCT |
E.6.9 | Dose response | Information not present in EudraCT |
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 | Yes |
E.7.1.3.1 | Other trial type description |
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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 | 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 | 18 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 1 |
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
|
End of Trial period is defined by completion of last patient follow up.
i.e. Period of patient recruitment 3 years, Period of treatment 6 months, Period of patient follow up 3 years
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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 | 6 |
E.8.9.1 | In the Member State concerned days | 0 |