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NCT05386680

Phase IIIb, Open-label, Single-arm, Multi-center Study to Evaluate the Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally (1.2 x 10^14 Vector Genomes) to Participants 2 to < 18 Years of Age With Spinal Muscular Atrophy (SMA) Who Have Discontinued Treatment With Nusinersen (Spinraza®) or Risdiplam (Evrysdi®)

Study Summary

This was a Phase IIIb open-label, single arm, multi-center study to evaluate the safety, tolerability and efficacy of OAV101B in participants with SMA aged 2 to \<18 years after the discontinuation of treatment with nusinersen or risdiplam. The study aimed to enroll approximately 28 participants across each of 2 age brackets (2 to \<6 years, and 6 to \<18 years).

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Interventions

OAV101GENETIC
Intrathecal administration of OAV101 at a dose of 1.2 x 10\^14 vector genomes, one time dose

Study Locations

FacilityCityStateCountry
Boston Childrens HospitalBostonMassachusettsUnited States
Child Hosp Of The Kings DaughtersNorfolkVirginiaUnited States
University of Wisconsin Madison Medical SchoolMadisonWisconsinUnited States
Novartis Investigative SiteParkvilleVictoriaAustralia
Novartis Investigative SiteLeuvenBelgium
Novartis Investigative SiteMontrealQuebecCanada
Novartis Investigative SiteBronFrance
Novartis Investigative SiteToulouseFrance
Novartis Investigative SiteRomaRMItaly
Novartis Investigative SiteKurumeFukuokaJapan
Novartis Investigative SiteShinjuku KuTokyoJapan
Novartis Investigative SiteUtrechtNetherlands
Novartis Investigative SiteBarcelonaCataloniaSpain

Official Trial Information

View on ClinicalTrials.gov

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