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NCT05089656

A Randomized, Sham-controlled, Double-blind Study to Evaluate the Efficacy and Safety of Intrathecal OAV101 in Type 2 Spinal Muscular Atrophy (SMA) Patients Who Are ≥ 2 to < 18 Years of Age, Treatment Naive, Sitting, and Never Ambulatory

Study Summary

This was a Phase III multi-center, single dose (1.2 x 10\^14 vector genomes), randomized, sham controlled, double-blind study that investigates the efficacy, safety and tolerability of OAV101B in treatment naive, sitting and never ambulatory SMA patients 2 to \<18 years of age.

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Interventions

OAV101GENETIC
Gene therapy
Sham controlPROCEDURE
The sham procedure will consist of a small needle prick on the lower back at the location where the LP injection is normally made. The needle will break the skin, but no needle insertion for lumbar puncture will occur.

Study Locations

FacilityCityStateCountry
Connecticut Children's Medical CenterFarmingtonConnecticutUnited States
Ann & Robert H. Lurie Children's Hospital of ChicagoChicagoIllinoisUnited States
Clinic for Special ChildrenStrasburgPennsylvaniaUnited States
St Jude Children's Research HospitalMemphisTennesseeUnited States
Child Hosp Of The Kings DaughtersNorfolkVirginiaUnited States
Children's Specialty Group/CHKDNorfolkVirginiaUnited States
Novartis Investigative SiteCuritibaParanáBrazil
Novartis Investigative SiteSão PauloSão PauloBrazil
Novartis Investigative SiteBeijingBeijing MunicipalityChina
Novartis Investigative SiteChongqingChongqing MunicipalityChina
Novartis Investigative SiteGuangzhouGuangdongChina
Novartis Investigative SiteShenzhenGuangdongChina
Novartis Investigative SiteChengduSichuanChina
Novartis Investigative SiteHangzhouZhejiangChina
Peking University First HospitalBeijingChina
Novartis Investigative SiteBeijingChina
Novartis Investigative SiteBeijingChina
Novartis Investigative SiteCopenhagenDenmark
Paediatric NeurologyCopenhagenDenmark
Sir Ganga Ram HospitalNew DelhiNational Capital Territory of DelhiIndia
Novartis Investigative SiteNew DelhiNational Capital Territory of DelhiIndia
Novartis Investigative SiteNew DelhiNational Capital Territory of DelhiIndia
AIIMS, Ansari NagarNew DelhiNew DelhiIndia
Novartis Investigative SiteKolkataWest BengalIndia
Novartis Investigative SiteHyderabadIndia
Novartis Investigative SiteMumbaiIndia
P.D. Hinduja National Hospital & MRCMumbaiIndia
Novartis Investigative SiteKuala LumpurMalaysia
Novartis Investigative SiteKuala LumpurMalaysia
Hospital Civil De Guadalajara Fray Antonio AlcaldeGuadalajaraJaliscoMexico
Novartis Investigative SiteGuadalajaraJaliscoMexico
Novartis Investigative SiteMexico CityMexico CityMexico
Novartis Investigative SiteRiyadhSaudi Arabia
Novartis Investigative SiteSingaporeSingapore
Novartis Investigative SiteCape TownSouth Africa
Red Cross War Memorial Childrens HospitalCape TownSouth Africa
Kaohsiung Medical University HospitalKaohsiung CityTaiwan
Novartis Investigative SiteKaohsiung CityTaiwan
Novartis Investigative SiteBangkokThailand
Siriraj HospitalBangkokThailand
National Children's HospitalHanoiVietnam
Novartis Investigative SiteHanoiVietnam

Official Trial Information

View on ClinicalTrials.gov

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