Une étude d'innocuité à long terme chez des patients brésiliens ayant reçu un diagnostic d'amyotrophie spinale traités par Zolgensma (ARISER)
Une étude d'innocuité à long terme chez des patients brésiliens avec un diagnostic confirmé d'amyotrophie spinale (AMS) traités par l'Onasemnogène Abeparvovec (Zolgensma®) - Étude ARISER
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Aperçu de l'étude
Statut
Les conditions
Intervention / Traitement
Description détaillée
This study is a non-interventional Post Authorization Safety Study (PASS) to evaluate long-term, real-world safety data of Brazilian pediatric patients diagnosed with SMA and treated with Onasemnogene Abeparvovec (Zolgensma®) for up to 15 years after the treatment. This study will support the benefit-risk assessment of Onasemnogene Abeparvovec in the approved indications and may also allow for detection of new safety signals and provide further guidance on the management of safety risks associated with Onasemnogene Abeparvovec to patients/caregivers, health care providers (HCPs) and treating physicians, as required by Brazilian Health Authority ANVISA as a conditional measure for granting Zolgensma®'s authorization.
Type d'étude
Inscription (Estimé)
Contacts et emplacements
Coordonnées de l'étude
- Nom: Novartis Pharmaceuticals
- Numéro de téléphone: +41613241111
- E-mail: novartis.email@novartis.com
Sauvegarde des contacts de l'étude
- Nom: Novartis Pharmaceuticals
Lieux d'étude
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Paraná
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Curitiba, Paraná, Brésil, 81520-060
- Recrutement
- Novartis Investigative Site
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São Paulo
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São Paulo, São Paulo, Brésil, 05403-000
- Recrutement
- Novartis Investigative Site
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Critères de participation
Critère d'éligibilité
Âges éligibles pour étudier
Accepte les volontaires sains
Sexes éligibles pour l'étude
Méthode d'échantillonnage
Population étudiée
The study population will consist of approximately 50 Brazilian pediatric patients with SMA who were treated with Onasemnogene Abeparvovec (Zolgensma®) in the commercial setting, the closed MAP, or the phase IV OFELIA trial. Patients will be recruited in up to 3 centers in Brazil, over one year of recruitment.
La description
Inclusion Criteria:
- Subject's parent or legal guardian has provided signed eICF.
- Subject with SMA, genetically confirmed: with a bi-allelic mutation in the SMN1 gene, and a clinical diagnosis of SMA Type 1 or up to 3 copies of the SMN2 gene.
- Subject treated* with Onasemnogene Abeparvovec (Zolgensma®) prior to enrolling in this study.
Subjects treated with nusinersen or risdiplam prior to Onasemnogene Abeparvovec (Zolgensma®) can be enrolled if currently not receiving it.
*Subjects can be enrolled in this study on the day treated with Onasemnogene Abeparvovec (Zolgensma®) or if prior medical history is available to complete all assessments retrospectively, in accordance with local ethical requirements.
- Subject and parent/guardian are willing and able to comply with the phone contacts through the course of the study
Exclusion Criteria:
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Patients currently enrolled in any interventional clinical trial** other than the phase IV OFELIA trial will be excluded from the study.
- Subjects who were enrolled in a clinical trial (independently of the disease indication and interventional treatment) but are not currently enrolled, can be included in this study.
During the follow-up, subjects who enroll any clinical trial with pharmacological intervention will discontinue from this study.
Plan d'étude
Comment l'étude est-elle conçue ?
Détails de conception
- Modèles d'observation: COHORT
- Perspectives temporelles: OTHER
Cohortes et interventions
Groupe / Cohorte |
Intervention / Traitement |
|---|---|
|
Spinal Muscular Atrophy Patients
Brazilian pediatric patients with a confirmed diagnosis of Spinal Muscular Atrophy treated with Onasemnogene Abeparvovec |
Retrospective and prospective observational study. There is no treatment allocation.
Autres noms:
|
Que mesure l'étude ?
Principaux critères de jugement
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Incidence and severity of treatment-emergent SAEs
Délai: Up to 5 years
|
Incidence and severity of treatment-emergent Serious AEs (SAEs) |
Up to 5 years
|
Mesures de résultats secondaires
Mesure des résultats |
Description de la mesure |
Délai |
|---|---|---|
|
Number of patients who experience at least one AESI and number of patients by AESI
Délai: Up to 15 years
|
Adverse Event of Special Interest: thrombocytopenia, hepatotoxicity, thrombotic microangiopathy (TMA), dorsal root ganglia toxicity, cardiac AEs, new malignancies, new incidence of neurologic, autoimmune, and hematologic disorder |
Up to 15 years
|
|
Number of patients who have survived and have not required permanent ventilatory assistance
Délai: Up to 15 years
|
Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation. |
Up to 15 years
|
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Time until death or the need for permanent ventilatory assistance
Délai: Up to 15 years
|
Evaluation of permanent ventilatory assistance, defined as requiring invasive ventilation(tracheostomy), or respiratory assistance for 16 or more hours per day (including non-invasiveventilatory support) continuously for 14 or more days in the absence of an acute reversible illness,excluding perioperative ventilation. |
Up to 15 years
|
|
Number of patients who achieve each Developmental Motor Milestone
Délai: Up to 15 years
|
Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone. |
Up to 15 years
|
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Number of patients who achieve each WHO Developmental Milestone within age percentiles
Délai: Up to 15 years
|
Evaluation of WHO Developmental Milestones: sitting without support, hands, and knees crawling, standing with assistance, walking with assistance, standing alone, and walking alone. |
Up to 15 years
|
Collaborateurs et enquêteurs
Parrainer
Les enquêteurs
- Directeur d'études: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Dates d'enregistrement des études
Dates principales de l'étude
Début de l'étude (Réel)
Achèvement primaire (Estimé)
Achèvement de l'étude (Estimé)
Dates d'inscription aux études
Première soumission
Première soumission répondant aux critères de contrôle qualité
Première publication (Réel)
Mises à jour des dossiers d'étude
Dernière mise à jour publiée (Réel)
Dernière mise à jour soumise répondant aux critères de contrôle qualité
Dernière vérification
Plus d'information
Termes liés à cette étude
Termes MeSH pertinents supplémentaires
Autres numéros d'identification d'étude
- COAV101ABR01
Plan pour les données individuelles des participants (IPD)
Prévoyez-vous de partager les données individuelles des participants (DPI) ?
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