En langsiktig sikkerhetsstudie hos brasilianske pasienter med diagnose av spinal muskelatrofi behandlet med Zolgensma (ARISER)
En langsiktig sikkerhetsstudie i brasilianske pasienter med en bekreftet diagnose av spinal muskelatrofi (SMA) behandlet med Onasemnogen Abeparvovec (Zolgensma®) - ARISER-studie
A long-term safety study in Brazilian patients with a confirmed diagnosis of Spinal Muscular Atrophy (SMA) treated with Onasemnogene Abeparvovec (Zolgensma®)
Studieoversikt
Status
Forhold
Intervensjon / Behandling
Detaljert beskrivelse
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.
Studietype
Registrering (Antatt)
Kontakter og plasseringer
Studiekontakt
- Navn: Novartis Pharmaceuticals
- Telefonnummer: +41613241111
- E-post: novartis.email@novartis.com
Studer Kontakt Backup
- Navn: Novartis Pharmaceuticals
Studiesteder
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Paraná
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Curitiba, Paraná, Brasil, 81520-060
- Rekruttering
- Novartis Investigative Site
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São Paulo
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São Paulo, São Paulo, Brasil, 05403-000
- Rekruttering
- Novartis Investigative Site
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Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Prøvetakingsmetode
Studiepopulasjon
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.
Beskrivelse
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.
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Observasjonsmodeller: COHORT
- Tidsperspektiver: OTHER
Kohorter og intervensjoner
Gruppe / Kohort |
Intervensjon / Behandling |
|---|---|
|
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.
Andre navn:
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Incidence and severity of treatment-emergent SAEs
Tidsramme: Up to 5 years
|
Incidence and severity of treatment-emergent Serious AEs (SAEs) |
Up to 5 years
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
|---|---|---|
|
Number of patients who experience at least one AESI and number of patients by AESI
Tidsramme: 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
Tidsramme: 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
|
|
Time until death or the need for permanent ventilatory assistance
Tidsramme: 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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Number of patients who achieve each Developmental Motor Milestone
Tidsramme: 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
|
|
Number of patients who achieve each WHO Developmental Milestone within age percentiles
Tidsramme: 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
|
Samarbeidspartnere og etterforskere
Sponsor
Etterforskere
- Studieleder: Novartis Pharmaceuticals, Novartis Pharmaceuticals
Studierekorddatoer
Studer hoveddatoer
Studiestart (Faktiske)
Primær fullføring (Antatt)
Studiet fullført (Antatt)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Faktiske)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Ytterligere relevante MeSH-vilkår
Andre studie-ID-numre
- COAV101ABR01
Plan for individuelle deltakerdata (IPD)
Planlegger du å dele individuelle deltakerdata (IPD)?
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