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

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®)

• Status: Completed
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 27 (actual)
• Sponsor: Novartis Pharmaceuticals · Industry
• Sex: All
• Age: 2 Years – 17 Years
• Healthy volunteers: Not accepted

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).

Detailed description

Eligible participants received a single OAV101B administration of 1.2x1014 vector genomes on Day 1 (Treatment period) and were followed for a period of 52 weeks. Participants were admitted to the hospital on Day -1 for pre-treatment baseline procedures. After receiving OAV101B on Day 1, participants underwent in-patient safety monitoring over the next 48 hours, after which the participant could be discharged, based on Investigator judgment.

Conditions

• Spinal Muscular Atrophy

Interventions

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

Timeline

• Start date: 2023-01-12
• Primary completion: 2024-11-29
• Completion: 2024-11-29
• First posted: 2022-05-23
• Last updated: 2026-01-13
• Results posted: 2025-06-03

Locations

13 sites across 9 countries: United States, Australia, Belgium, Canada, France, Italy, Japan, Netherlands, Spain

Regulatory

• FDA-regulated drug study