E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
De novo allograft transplantation (liver, heart, kidney) in paediatric patients. |
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MedDRA Classification |
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 principal objective is to find out how much of Modigraf is absorbed and used in the body and how fast it leaves the body. This is called pharmacokinetics (PK). The results will then help to decide how much Modigraf in future should be given to children and young people following transplantation. |
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E.2.2 | Secondary objectives of the trial |
Secondary objective of this study is gathering information on the safety and efficacy of Modigraf. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. The subject is 12 years of age or younger. 2. The subject is the recipient of a solid organ (liver, kidney or heart) transplant. Multiorgan transplants are acceptable as long as one of the organs transplanted is liver, kidney or heart. 3. The subject’s parent(s) or their legal representative(s) has been fully informed and has given written informed consent to participate in the study. The subject has given assent where applicable. |
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E.4 | Principal exclusion criteria |
1. The subject has previously received another organ transplant (including liver, kidney or heart re-transplantation). 2. Subject has a high immunological risk, defined as a Panel Reactive Antibody (PRA) score >50% in the previous 6 months (only applicable for renal transplant recipients). 3. Cold ischemia time of the donor kidney greater than 30 hours (only applicable for renal transplant recipients). 4. Subject receives an AB0 incompatible donor organ. 5. Subject has significant renal impairment, defined as having serum creatinine ≥230 μmol/l (≥2.6 mg/dl) pre-transplantation (not applicable for renal transplant recipients). 6. Subject has significant liver disease, defined as having elevated ALT and/or AST and/or Total Bilirubin levels 3 times the upper value of the normal range during the 28 days prior to transplantation (not applicable for liver transplant recipients). 7. Subject with pulmonary vascular resistance greater than 4 Wood units which is unresponsive to treatment. 8. Subjects with malignancies or a history of malignancy within the last 5 years. 9. Subject has a significant, uncontrolled systemic infection and/or severe diarrhea, vomiting, active upper gastrointestinal disorder that may affect the absorption of tacrolimus or has an active peptic ulcer. 10. Subject requires systemic immunosuppressive medication for any indication other than transplantation. 11. Recipient or donor known to be HIV, HCV or HBV positive. 12. Known allergy or intolerance to steroids, macrolide antibiotics, basiliximab or tacrolimus. 13. Subject is currently participating in another clinical trial and/or has been taking an investigational drug in the 3 months prior to transplantation. 14. Subject is unlikely to comply with the visits scheduled in the protocol. 15. Subjects taking or requiring to be treated with medication or substances prohibited by the study protocol. |
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E.5 End points |
E.5.1 | Primary end point(s) |
PK parameters for tacrolimus will be determined after the morning dose on Day 1 and Day 7 (+7 days): - AUC tau - C max - t max - C trough Other PK parameters may be calculated where appropriate. |
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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 | Yes |
E.6.3 | Therapy | No |
E.6.4 | Safety | Yes |
E.6.5 | Efficacy | Yes |
E.6.6 | Pharmacokinetic | Yes |
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) | No |
E.7.4 | Therapeutic use (Phase IV) | Yes |
E.8 Design of the trial |
E.8.1 | Controlled | No |
E.8.1.1 | Randomised | Information not present in EudraCT |
E.8.1.2 | Open | Information not present in EudraCT |
E.8.1.3 | Single blind | Information not present in EudraCT |
E.8.1.4 | Double blind | Information not present in EudraCT |
E.8.1.5 | Parallel group | Information not present in EudraCT |
E.8.1.6 | Cross over | Information not present in EudraCT |
E.8.1.7 | Other | Information not present in EudraCT |
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.8.4.1 | Number of sites anticipated in Member State concerned | 3 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 8 |
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
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Last visit of last patient. |
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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 | 2 |
E.8.9.1 | In the Member State concerned months | 0 |
E.8.9.1 | In the Member State concerned days | 0 |
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 |
E.8.9.2 | In all countries concerned by the trial days | 0 |