MedTrace Logo

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 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 40

Last updated 2024-02-22

No results posted yet for this study

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.

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.

Sponsors & Collaborators

  • Biocad

    lead INDUSTRY

Principal Investigators

  • Arina V Zinkina-Orikhan, PhD · Director of Clinical Development Department, BIOCAD

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Max Age
240 Days
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2023-02-02
Primary Completion
2025-09-30
Completion
2030-08-31

Countries

  • Belarus
  • Russia

Study Locations

More Related Trials

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT05747261 on ClinicalTrials.gov