E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Poliquistosis Renal Autosómica Dominante (PQRAD) |
|
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 |
Evaluar la eficacia, seguridad y tolerabilidad de Rapamicina para retrasar y/o enlentecer el crecimiento de los quistes y del volumen renal y para prevenir la pérdida de la función renal en pacientes con PQRAD con función renal conservada, midiendo los volúmenes de los riñones por RMN y la función renal durante un periodo de 1 año. |
|
E.2.2 | Secondary objectives of the trial |
Monitorizar la evolución de la presión arterial cada 3 meses. Monitorizar la aparición de proteinuria cada 3 meses. Analizar la evolución de los quistes hepáticos a lo largo del estudio. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Edad igual o mayor de 18 años. 2. Pacientes con antecedentes familiares de PQRAD y/o diagnóstico confirmado de PQRAD tipo1. 3. Serán incluidos los pacientes con una Tasa de Filtración Glomerular (aclaramiento de creatinina) > 70 ml/min. 4. Haber dado su consentimiento informado por escrito. |
|
E.4 | Principal exclusion criteria |
1. Embarazo y lactancia. 2. Pacientes con aclaramiento de creatinina < de 70 ml/min. 3. Pacientes con proteinuria > 500 mg/ 24 h. 4. Administración conjunta de medicamentos inductores o inhibidores del CYP 3A4. 5. Hiperlipemia. 6. Infección severa en el momento de entrar al estudio. 7. Hipersensibilidad a la Rapamicina conocida. 8. Pacientes en quienes no se haya documentado, a través de técnicas de imagen, crecimiento del volumen renal en los 6 meses previos a la aleatorización. 9. Antecedentes de neoplasias en los últimos 2 años excepto cáncer de cérvix o cáncer cutáneo tratado. 10. Participación en otro ensayo clínico. |
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E.5 End points |
E.5.1 | Primary end point(s) |
Para evaluar la eficacia de la Rapamicina se comparará el porcentaje de variación del volumen renal (lo mismo hepático) a los 12 meses entre ambos grupos. |
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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 | 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 | 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 | Information not present in EudraCT |
E.7.1.2 | Bioequivalence study | Information not present in EudraCT |
E.7.1.3 | Other | Information not present in EudraCT |
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 | 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 | Yes |
E.8.2.3.1 | Comparator description |
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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 | 5 |
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 |