NCT05386680 - Phase IIIb, Open-label, Multi-center Study to Evaluate Safety, Tolerability and Efficacy of OAV101 Administered Intrathecally to Participants With SMA Who Discontinued Treatment With Nusinersen or Risdiplam | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

January 12, 2023

Primary Completion Date

November 29, 2024

Study Completion Date

November 29, 2024

Conditions

Spinal Muscular Atrophy

Interventions

GENETIC

OAV101

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

Trial Locations (13)

Novartis Investigative Site, Leuven

Novartis Investigative Site, Parkville

Novartis Investigative Site, Utrecht

Child Hosp Of The Kings Daughters, Norfolk

Novartis Investigative Site, Toulouse

Novartis Investigative Site, Bron

Boston Childrens Hospital, Boston

University of Wisconsin Madison Medical School, Madison

Novartis Investigative Site, Montreal

Novartis Investigative Site, Roma

Novartis Investigative Site, Kurume

Novartis Investigative Site, Shinjuku Ku

Novartis Investigative Site, Barcelona