● Recruiting

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)

Condition: Spinal Muscular Atrophy (SMA) · Sponsor: Biocad

PhasePhase 1/Phase 2

Planned participants40

Who can joinAll sexes, N/A to 240 Days

Healthy volunteersNo

About this study

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.

This description comes directly from the study's public registry record.

Talk to the study team

Maria Morozova, MD, PhD · +7 (985) 910 28 13 · morozovama@biocad.ru

Always discuss trial participation with your own doctor first.

Locations (7)

1. State Institution Republican Scientific and Practical Center "Mother and Child"	Minsk, Belarus	Recruiting
Federal State Autonomous Educational Institution of Higher Education "N.I. Pirogov Russian National Research Medical University", Ministry of Health of the Russian Federation	Moscow, Russia	Recruiting
Federal State Autonomous Educational Institution of Higher Education "N.I. Pirogov Russian National Research Medical University", Ministry of Health of the Russian Federation	Moscow, Russia	Recruiting
Federal State Autonomous Institution "National Medical Research Center for Children's Health", Ministry of Health of the Russian Federation	Moscow, Russia	Recruiting
Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University", Ministry of Health of the Russian Federation	Saint Petersburg, Russia	Recruiting
Federal State Budgetary Institution "V. A. Almazov National Medical Research Center" of the Ministry of Health of the Russian Federation	Saint Petersburg, Russia	Recruiting
State Autonomous Healthcare Institution of the Sverdlovsk Region "Regional Children's Clinical Hospital"	Yekaterinburg, Russia	Recruiting

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Source record: clinicaltrials.gov/study/NCT05747261