Uno studio sulla sicurezza a lungo termine in pazienti brasiliani con diagnosi di atrofia muscolare spinale trattati con Zolgensma (ARISER)
Uno studio sulla sicurezza a lungo termine in pazienti brasiliani con diagnosi confermata di atrofia muscolare spinale (SMA) trattati con Onasemnogene Abeparvovec (Zolgensma®) - Studio ARISER
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
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.
Tipo di studio
Iscrizione (Stimato)
Contatti e Sedi
Contatto studio
- Nome: Novartis Pharmaceuticals
- Numero di telefono: +41613241111
- Email: novartis.email@novartis.com
Backup dei contatti dello studio
- Nome: Novartis Pharmaceuticals
Luoghi di studio
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Paraná
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Curitiba, Paraná, Brasile, 81520-060
- Reclutamento
- Novartis Investigative Site
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São Paulo
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São Paulo, São Paulo, Brasile, 05403-000
- Reclutamento
- Novartis Investigative Site
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Metodo di campionamento
Popolazione di studio
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.
Descrizione
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.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Modelli osservazionali: COHORT
- Prospettive temporali: OTHER
Coorti e interventi
Gruppo / Coorte |
Intervento / Trattamento |
|---|---|
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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.
Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Incidence and severity of treatment-emergent SAEs
Lasso di tempo: Up to 5 years
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Incidence and severity of treatment-emergent Serious AEs (SAEs) |
Up to 5 years
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
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Number of patients who experience at least one AESI and number of patients by AESI
Lasso di tempo: 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
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Number of patients who have survived and have not required permanent ventilatory assistance
Lasso di tempo: Up to 15 years
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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
Lasso di tempo: Up to 15 years
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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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Number of patients who achieve each Developmental Motor Milestone
Lasso di tempo: Up to 15 years
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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
Lasso di tempo: Up to 15 years
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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
|
Collaboratori e investigatori
Sponsor
Investigatori
- Direttore dello studio: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Studiare le date dei record
Studia le date principali
Inizio studio (Effettivo)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- COAV101ABR01
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