Actively 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)
Led by Biocad · Updated on 2024-02-22
40
Participants Needed
7
Research Sites
391 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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
Official Title
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)
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Signed informed consent by the child's legal representative
- Child under 240 days old at the time of consent
- Diagnosis of 5q-SMA confirmed by genetic testing with 2 or 3 copies of the SMN2 gene
- For children with 2 copies of SMN2: presymptomatic or symptomatic with disease onset before 180 days of age
- For children with 3 copies of SMN2: symptomatic SMA type 1 with disease onset before 180 days of age
- Legal representative able to understand study information and follow procedures
You will not qualify if you...
- Child diagnosed with HIV, hepatitis B, hepatitis C, or congenital syphilis, or mother with documented HIV infection
- Legal representative unwilling to use alternative feeding methods if swallowing disorders occur
- Anti-AAV9 antibody titer greater than 1:50 (may retest to confirm eligibility)
- Need for respiratory support 16 hours or more per day or tracheostomy
- Previous or planned treatment with nusinersen, risdiplam, branaplam, onasemnogene abeparvovec, or other SMN2 splicing modifiers or AAV-based gene therapies within 12 months
- Need for myopathy or neuropathy treatment drugs, diabetes drugs, or immunosuppressive therapy (except for certain pre/post-medications)
- Abnormal lab results at screening including elevated liver enzymes, bilirubin, creatinine, abnormal hemoglobin, high white blood cell count, or elevated Troponin I
- Any other disease affecting safety or study outcomes as judged by the investigator
- Acute or chronic liver failure
- Known allergy or intolerance to study drug components or related medications
- Participation in other clinical studies or prior experimental therapy studies
AI-Screening
AI-Powered Screening
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Trial Site Locations
Total: 7 locations
1
1. State Institution Republican Scientific and Practical Center "Mother and Child"
Minsk, Belarus
Actively Recruiting
2
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, 117997
Actively Recruiting
3
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, 117997
Actively Recruiting
4
Federal State Autonomous Institution "National Medical Research Center for Children's Health", Ministry of Health of the Russian Federation
Moscow, Russia, 119991
Actively Recruiting
5
Federal State Budgetary Educational Institution of Higher Education "St. Petersburg State Pediatric Medical University", Ministry of Health of the Russian Federation
Saint Petersburg, Russia, 194100
Actively Recruiting
6
Federal State Budgetary Institution "V. A. Almazov National Medical Research Center" of the Ministry of Health of the Russian Federation
Saint Petersburg, Russia, 197341
Actively Recruiting
7
State Autonomous Healthcare Institution of the Sverdlovsk Region "Regional Children's Clinical Hospital"
Yekaterinburg, Russia, 620149
Actively Recruiting
Research Team
M
Maria Morozova, MD, PhD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
3
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