E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
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MedDRA Classification |
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 |
Outcome assessment at the end of the study The primary end point is measurement of kidney function by the estimated glomerular filtration rate and extent of proteinuria as 24h urinary excretion and fractional protein excretion. The results will be compared with the change of eGFR during the preceeding six months, and the mean of two protein excretion measurements at study entry. Progresssion of disease is defined as a significant more rapid decline of eGFR compared to pretreatment values, or a significant higher protein excretion. Improvement of both markers is defined as improvement of the disease, and no change as disease beeing stable.
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E.2.2 | Secondary objectives of the trial | |
E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Biopsy proven renal amyoidosis 2. Serum Amyloid A concentration greater than 25mg/l 3. Estimated GFR between 30 to 90ml/min 4. Men or women aged >18years 5. Provision of informed consent
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E.4 | Principal exclusion criteria |
1. Estimated GFR < 30ml/min 2. Kidney disease other than renal amyloidosis 3. Child bearing potential with lack of a reliable method of contraception. 4. Poor compliance 5. Medical history that might limit the individual’s ability to take the trial treatment for the duration of the study. 6. Simultanous administration of TNF-blocking agents (etanercept, adalimumab, and infliximab)
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E.5 End points |
E.5.1 | Primary end point(s) |
The primary end point is measurement of kidney function by the estimated glomerular filtration rate and extent of proteinuria as 24h urinary excretion and fractional protein excretion. The results will be compared with the change of eGFR during the preceeding six months, and the mean of two protein excretion measurements at study entry. Progresssion of disease is defined as a significant more rapid decline of eGFR compared to pretreatment values, or a significant higher protein excretion. Improvement of both markers is defined as improvement of the disease, and no change as disease beeing stable. |
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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 | No |
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 | Information not present in EudraCT |
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 | 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) | 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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
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 | Information not present in EudraCT |
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.8.9.1 | In the Member State concerned years | 3 |
E.8.9.1 | In the Member State concerned months | |
E.8.9.1 | In the Member State concerned days | |