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drugset / Trial / NCT05747261

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)

NCT05747261 ↗

Phase 1/2 Recruiting 40 enrolled Biocad
Non-randomizedSequentialOpen-labelTreatment

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.

Timeline

Start
2023-02-02
Primary completion
2025-09
Completion
2030-08

Drugs

EvaluationDrugModalityDoseRoute
Subject ANB-004 Gene therapy (AAV / viral vector) — Intravenous

Indications

spinal muscular atrophy