E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
El déficit de alfa-1-antitripsina (AAT) es una enfermedad hereditaria de expresión autosómica recesiva que se caracteriza por concentraciones anormalmente reducidas de AAT en sangre, lo que comporta en su forma homocigótica, un elevado riesgo de aparición de enfisema pulmonar precoz, y en determinadas ocasiones enfermedad hepática (1). |
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MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 9 |
E.1.2 | Level | PT |
E.1.2 | Classification code | 10001806 |
E.1.2 | Term | Alpha-1 anti-trypsin deficiency |
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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 |
Validar la idoneidad de las pautas de tratamiento con intervalos ampliados obtenidas en el modelo farmacocinético previo mediante el estudio farmacodinámico de la actividad antiinflamatoria de la AAT a nivel bronquial. |
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E.2.2 | Secondary objectives of the trial |
1 Evaluar las concentraciones de AAT y de marcadores de inflamación bronquial (variables de estudio) en estado de equilibrio estacionario en secreciones bronquiales y en suero, para un régimen posológico de AAT que ha demostrado mantener las concentraciones séricas de AAT > 0.5 g/L durante el 85% del intervalo terapéutico (D2185). 2 Correlacionar las concentraciones de AAT y las de marcadores de inflamación bronquial (actividad anti-elastasa, mieloperoxidasa, IL6, IL8 y LTB4) en muestras de esputo inducido y suero en diferentes momentos del intervalo entre dosis de AAT. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
Se incluirán todos los pacientes adultos controlados en el Hospital Clínic de Barcelona con diagnóstico de déficit de AAT y que cumplan los criterios aceptados de terapia sustitutiva con AAT exógena (normativa SEPAR ? Grupo de trabajo Déficit AAT). En la actualidad, en nuestro centro siguen tratamiento sustitutivo 8 pacientes con déficit grave de AAT. |
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E.4 | Principal exclusion criteria |
Criterios de exclusión: - Tabaquismo activo - Hipersensibilidad demostrada a la administración de AAT exógena - Déficit de IgA |
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E.5 End points |
E.5.1 | Primary end point(s) |
Se determinarán en muestras de sangre y secreciones bronquiales: - concentraciones de AAT (nefelometría) - actividad anti-elastasa - mieloperoxidasa - marcadores de inflamación: IL6, IL8 y LTB4 Se harán determinaciones de los valores séricos de Proteína C reactiva |
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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 | No |
E.6.5 | Efficacy | No |
E.6.6 | Pharmacokinetic | No |
E.6.7 | Pharmacodynamic | Yes |
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 | No |
E.8.1.1 | Randomised | No |
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 | No |
E.8.1.6 | Cross over | No |
E.8.1.7 | Other | Yes |
E.8.1.7.1 | Other trial design description |
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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
| Yes |
E.8.4 | The trial involves multiple sites in the Member State concerned | No |
E.8.4.1 | Number of sites anticipated in Member State concerned | 1 |
E.8.5 | The trial involves multiple Member States | No |
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
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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 | |
E.8.9.1 | In the Member State concerned months | 12 |
E.8.9.1 | In the Member State concerned days | |