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)

PHASE1/PHASE2RecruitingINTERVENTIONAL
Enrollment

40

Participants

Timeline

Start Date

February 2, 2023

Primary Completion Date

September 30, 2025

Study Completion Date

August 31, 2030

Conditions
Spinal Muscular Atrophy (SMA)
Interventions
GENETIC

ANB-004, dose 1

Adeno-associated viral vector carrying the SMN gene single infusion at dose 1. The duration of the infusion is about 60 minutes.

GENETIC

ANB-004, dose 2

Adeno-associated viral vector carrying the SMN gene single infusion at dose 2. The duration of the infusion is about 60 minutes.

GENETIC

ANB-004, dose 3

Adeno-associated viral vector carrying the SMN gene single infusion at dose 3. The duration of the infusion is about 60 minutes.

Trial Locations (7)

117997

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

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

119991

RECRUITING

"Federal State Autonomous Institution National Medical Research Center for Children's Health, Ministry of Health of the Russian Federation", Moscow

194100

RECRUITING

"Federal State Budgetary Educational Institution of Higher Education St. Petersburg State Pediatric Medical University, Ministry of Health of the Russian Federation", Saint Petersburg

197341

RECRUITING

"Federal State Budgetary Institution V. A. Almazov National Medical Research Center of the Ministry of Health of the Russian Federation", Saint Petersburg

620149

RECRUITING

"State Autonomous Healthcare Institution of the Sverdlovsk Region Regional Children's Clinical Hospital", Yekaterinburg

Unknown

RECRUITING

"1. State Institution Republican Scientific and Practical Center Mother and Child", Minsk

Sponsors

Lead Sponsor

All Listed Sponsors
lead

Biocad

INDUSTRY

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) | Biotech Hunter | Biotech Hunter