E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Familial Amyloid Polyneuropathy |
Polineuropatía amiloide familiar |
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E.1.1.1 | Medical condition in easily understood language |
Familial Amyloid Polyneuropathy |
Polineuropatía amiloide familiar |
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E.1.1.2 | Therapeutic area | Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] |
MedDRA Classification |
E.1.2 Medical condition or disease under investigation |
E.1.2 | Version | 20.0 |
E.1.2 | Level | LLT |
E.1.2 | Classification code | 10057949 |
E.1.2 | Term | Familial amyloid polyneuropathy |
E.1.2 | System Organ Class | 100000004850 |
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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 |
To monitor the survival of patients who are no longer actively participating in ISIS 420915-CS2 or ISIS 420915-CS3 and were in the ISIS 420915-CS2 safety population (defined as all patients that received at least 1 dose of Study Drug (placebo or inotersen)) |
Comprobar la supervivencia de pacientes que ya no participan de forma activa en ISIS 420915-CS2 o ISIS 420915-CS3 y que se encontraban en la población de seguridad de ISIS 420915-CS2 (definida como todos los pacientes que recibieron, al menos, 1 dosis del fármaco del estudio [placebo o inotersén]). |
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E.2.2 | Secondary objectives of the trial |
To collect information about the cause of death and related circumstances |
Recopilar información acerca de la causa de la muerte y de las circunstancias relacionadas. |
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E.2.3 | Trial contains a sub-study | No |
E.3 | Principal inclusion criteria |
1. Patient or representative must have given written informed consent (signed and dated) and any authorizations required by local law and be able to comply with all study requirements.
2. Patient must be part of the ISIS 420915-CS2 safety population (defined as all patients that received at least 1 dose of Study Drug (placebo or inotersen)) or representative(s) of patient in ISIS 420915-CS2 safety population |
1. El paciente o su representante deben haber otorgado su consentimiento informado por escrito (firmado y fechado) y cualquier autorización exigida por la legislación local, además de ser capaces de cumplir con todos los requisitos del estudio. 2. Población de seguridad de ISIS 420915-CS2 (definida como todos los pacientes que recibieron, al menos, 1 dosis del fármaco del estudio [placebo o inotersén]) o representante(s) del paciente de la población de seguridad de ISIS 420915-CS2. |
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E.4 | Principal exclusion criteria |
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E.5 End points |
E.5.1 | Primary end point(s) |
- Time to death from first dose of Study Drug in ISIS 420915-CS2 (inotersen or placebo) - Time to death from first dose of inotersen - Percentage of patients that died within 66 weeks, as well as 3 years, of first dose of Study Drug in ISIS 420915-CS2 - Percentage of patients whose survival status is unknown 66 weeks, as well as 3 years, after first dose of Study Drug in ISIS 420915-CS2 |
-Tiempo transcurrido hasta la muerte desde la toma de la primera dosis del fármaco del estudio en ISIS 420915-CS2 (inotersen o placebo). -Tiempo transcurrido hasta la muerte desde la toma de la primera dosis de inotersen. -Porcentaje de pacientes que murieron en las 66 semanas y en los 3 años posteriores a tomar la primera dosis del fármaco del estudio en ISIS 420915-CS2. -Porcentaje de pacientes cuyo estado de supervivencia se desconoce a las 66 semanas y a los 3 años después de la primera dosis del fármaco del estudio en ISIS 420915-CS2. |
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E.5.1.1 | Timepoint(s) of evaluation of this end point |
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E.5.2 | Secondary end point(s) |
- Cause of death |
- Causa de la muerte |
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E.5.2.1 | Timepoint(s) of evaluation of this end point |
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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 | No |
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 | 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) | Yes |
E.7.4 | Therapeutic use (Phase IV) | No |
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 |
Seguimiento del paciente para evaluar su supervivencia |
Patient follow-up to determine survival status |
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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 | 2 |
E.8.5 | The trial involves multiple Member States | Yes |
E.8.5.1 | Number of sites anticipated in the EEA | 10 |
E.8.6 Trial involving sites outside the EEA |
E.8.6.1 | Trial being conducted both within and outside the EEA | Yes |
E.8.6.2 | Trial being conducted completely outside of the EEA | No |
E.8.6.3 | If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned |
Argentina |
Brazil |
France |
Germany |
Italy |
New Zealand |
Portugal |
Spain |
United Kingdom |
United States |
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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
|
Last attempt by the study center, in the last participating country, to contact the patient and/or patient’s representative(s) to determine survival status and, if applicable, the circumstances related to death.
LVLS is not applicable in this trial since subjects will not attend site visits. |
El ultimo intento por el centro del studio, en el ulimo pais particopante, para contactar el paciente y/o el(los) representante (s) del paciente para determinar el estado de supervivencia y, si procede, las circunstancias relacionadas con la muerte.
Ultima visita del ultimo paciente no aplica en este studio ya que los pacientes no asistiran a visitas en el centro. |
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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 | 2 |
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 |