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NCT05335876

Long-term Follow-up of Patients With Spinal Muscular Atrophy Treated With OAV101 IT or OAV101 IV in Clinical Trials

Study Summary

This is a global, prospective, multi-center study that is designed to assess the long-term safety and efficacy of OAV101 in patients who participated in an OAV101 clinical trial. The assessments of safety and efficacy in Study COAV101A12308 will continue for 5 years after enrollment in this study.

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Interventions

onasemnogene abeparvovecBIOLOGICAL
Onasemnogene abeparvovec is a non-replicating recombinant adeno-associated virus serotype 9 containing the human survival motor neuron gene under the control of the ytomegalovirus enhancer/chicken β-actin-hybrid promoter. Onasemnogene abeparvovec is administered as a one-time intravenous (IV) infusion or intrathecal (IT) injection. Dosage determined by participant weight.

Study Locations

FacilityCityStateCountry
Child Hosp Of The Kings DaughtersNorfolkVirginiaUnited States
Novartis Investigative SiteSydneyNew South WalesAustralia
Novartis Investigative SiteLeuvenBelgium
Novartis Investigative SiteCuritibaParanáBrazil
Novartis Investigative SiteSão PauloSão PauloBrazil
Novartis Investigative SiteMontrealQuebecCanada
Novartis Investigative SiteChongqingChongqing MunicipalityChina
Novartis Investigative SiteGuangzhouGuangdongChina
Novartis Investigative SiteChengduSichuanChina
Novartis Investigative SiteBeijingChina
Novartis Investigative SiteBeijingChina
Novartis Investigative SiteBeijingChina
Novartis Investigative SiteCopenhagenDenmark
Novartis Investigative SiteBronFrance
Novartis Investigative SiteGarchesFrance
Novartis Investigative SiteStrasbourgFrance
Novartis Investigative SiteToulouseFrance
Novartis Investigative SiteRomaRMItaly
Novartis Investigative SiteKurumeFukuokaJapan
Novartis Investigative SiteShinjuku KuTokyoJapan
Novartis Investigative SiteKuala LumpurMalaysia
Novartis Investigative SiteKuala LumpurMalaysia
Novartis Investigative SiteUtrechtNetherlands
Novartis Investigative SiteRiyadhSaudi Arabia
Novartis Investigative SiteSingaporeSingapore
Novartis Investigative SiteBarcelonaSpain
Novartis Investigative SiteKaohsiung CityTaiwan
Novartis Investigative SiteTaipeiTaiwan
Novartis Investigative SiteBangkokThailand
Novartis Investigative SiteLondonUnited Kingdom
Novartis Investigative SiteNewcastle upon TyneUnited Kingdom
Novartis Investigative SiteHanoiVietnam

Official Trial Information

View on ClinicalTrials.gov

Data sourced from ClinicalTrials.gov. Last updated: April 14, 2026