E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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E.1.1.1 | Medical condition in easily understood language |
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E.1.1.2 | Therapeutic area | Body processes [G] - Immune system processes [G12] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 21.0 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10038533 |
E.1.2 | Term | Renal transplant |
E.1.2 | System Organ Class | 10042613 - Surgical and medical procedures |
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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 purpose of this study is to assess the pharmacokinetics (PK) efficacy and safety of belatacept in stable pediatric renal transplant recipients. |
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E.2.2 | Secondary objectives of the trial |
1. Safety Outcome Measures
2. Immunogenicity Measures
3. Pharmacodynamic Measures
4. Efficacy Measures
5. Exploratory |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1) 6-17 years old
2) Recipients of a renal allograft from a living donor or a deceased donor at least 6 months prior to enrollment.
3) Subject must be receiving a CNI-based (CsA [any formulation] or TAC) immunosuppressive regimen.
4) Subject must be receiving adjunctive background maintenance immunosuppression with MMF or EC-MPS/MPA.
5) Subject must be receiving a stable dose of corticosteroids
6) Subject must have stable estimated GFR ≥ 45 mL/min/1.73m2 (updated Schwartz formula)
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E.4 | Principal exclusion criteria |
1) EBV serostatus negative or unknown at time of transplant and screening
2) History of any treated or biopsy proven acute rejection (BPAR) within 3 months prior to randomization
3) Subjects who have experienced more than 1 episode of AR of the current allograft or any antibodymediated AR
4) Significant proteinuria
5) Active infection |
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E.5 End points |
E.5.1 | Primary end point(s) |
The PK of belatacept will be derived from serum concentration versus time data in pediatric subjects with stable renal transplant |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
At least 54 subjects have reached 6 months in multiple dose phase |
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E.5.2 | Secondary end point(s) |
1. Safety Outcome Measures
2. Immunogenicity Measures
3. Pharmacodynamic Measures
4. Efficacy Measures
5. Exploratory |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
1. At 12, 24 and 36 months
2. Day 1, every 6 months, at time of acute rejection, after discontinuation of study drug and at 8, 12 and 24 months post discontinuation of study drug.
3. Day 1, Month 1, 6 and 12
4. at 3, 6, 12, 24 and 36 months
5. at baseline, 6, 12, 24 and 36 months; Anti-viral T cell memory response at Day1, Day29 and 6 and 12 months |
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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 | No |
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) | 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 | 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 | No |
E.8.2 | Comparator of controlled trial |
E.8.2.1 | Other medicinal product(s) | Yes |
E.8.2.2 | Placebo | No |
E.8.2.3 | Other | No |
E.8.2.4 | Number of treatment arms in the trial | 2 |
E.8.3 |
Will this trial be conducted at a single site globally?
| No |
E.8.4 | Will this trial be conducted at multiple sites globally? | Yes |
E.8.6 Trial involving sites outside the EEA |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | Specify the countries outside of the EEA in which trial sites are planned |
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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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E.8.9 Initial estimate of the duration of the trial |
E.8.9.2 | In all countries concerned by the trial years | 5 |
E.8.9.2 | In all countries concerned by the trial months | 4 |
E.8.9.2 | In all countries concerned by the trial days | 0 |