E.1 Medical condition or disease under investigation |
E.1.1 | Medical condition(s) being investigated |
Familial Amyloid Polyneuropathy |
Polyneuropathie amyloïde familiale |
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E.1.1.1 | Medical condition in easily understood language |
Familial Amyloid Polyneuropathy |
Polyneuropathie amyloïde familiale |
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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)) |
Surveiller la survie des patients ne participant plus activement à ISIS 420915-CS2 ou ISIS 420915-CS3 et appartenant à la population de sécurité d'emploi de ISIS 420915-CS2 (tous les patients ayant reçu au moins une dose de médicament de l'étude [placebo ou inotersen]) |
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E.2.2 | Secondary objectives of the trial |
To collect information about the cause of death and related circumstances |
Recueillir des informations sur la cause du décès et les circonstances associées |
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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. Le patient ou son représentant doit avoir donné son consentement éclairé par écrit (signé et daté) ainsi que toutes les autorisations requises par la législation locale et être en mesure de se conformer à toutes les exigences de l’étude.
2. Le patient doit faire partie de la population de sécurité de l’étude ISIS 420915-CS2 (définie comme l’ensemble des patients qui ont reçu au moins 1 dose du médicament de l’étude [placebo ou inotersen]) ou un/des représentant(s) des patients de la population de sécurité de l’étude 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 |
- Délai de la première dose du médicament de l'étude au décès dans le cadre de l'étude ISIS 420915-CS2 (inotersen ou placebo)
- Délai de la première dose au décès d'inotersen
- Pourcentage de patients décédés dans les 66 semaines et 3 ans suivant la première dose de médicament de l'étude dans le cadre de l'étude ISIS 420915-CS2
- Pourcentage de patients dont le statut de survie est inconnu 66 semaines et 3 ans après la première dose du médicament de l'étude dans le cadre de l'étude 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) |
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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 |
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
| 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 | 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
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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. |
Dernière tentative du centre d’étude, dans le dernier pays participant, visant à contacter le patient et/ou son ou ses représentants afin de déterminer le statut de survie et, le cas échéant, les circonstances du décès.
La dernière visite du dernier patient ne s’applique pas à cette étude car les patients n’effectueront pas de visites aux centres. |
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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 |