Clinical Trial

Study of the Safety and Efficacy of an Adeno-Associated Viral Vector Carrying the SMN Gene After a Single Intravenous Administration of Escalating Doses in Children With Spinal Muscular Atrophy (BLUEBELL)

Study acronym: BLUEBELL
Recruiting Phase 1/2
View on ClinicalTrials.gov →
Record status
This record was last updated February 22, 2024 (before its estimated September 2025 completion). Its status may not reflect the trial's current state.
Summary
The goal of this multicenter, open-label, non-comparative, cohort study is to investigate the safety, immunogenicity, and efficacy of ANB-004 in children with spinal muscular atrophy. The study will have a standard 3+3 dose-escalation design.
Protocol Amendment History 4 amendments
This ClinicalTrials.gov record has been amended 4 times since 2023-02-17; most recent amendment 2024-02-21.
Trial Details
NCT Number NCT05747261
Lead Sponsor Biocad
Conditions Spinal Muscular Atrophy (SMA)
Enrollment 40 participants
Start Date 2023-02-02
Primary Completion 2025-09 (estimated)
Study Completion 2030-08 (estimated)
Updated on ClinicalTrials.gov 2024-02-22