Actively Recruiting
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
Led by Gemma Biotherapeutics · Updated on 2026-03-02
22
Participants Needed
1
Research Sites
168 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
GB221 is a gene therapy that delivers a working SMN1 gene to the motor neurons of people with spinal muscular atrophy (SMA) Type 1. This study will evaluate the safety, tolerability and efficacy of GB221 in two groups: 1. participants aged from 2 weeks to younger than 12 months presenting with symptoms of SMA Type 1 who have never received a treatment OR are receiving the drug risdiplam 2. participants aged from 2 weeks to younger than 5 months who are at risk of developing SMA Type 1 (presymptomatic) and have never received treatment OR are receiving the drug risdiplam.
CONDITIONS
Official Title
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of SMA Type 1 by gene mutation analysis with bi-allelic SMN1 mutations and up to 3 copies of SMN2 (for symptomatic participants)
- Age 2 weeks to younger than 12 months with disease onset during first 6 months of life (for symptomatic participants)
- At risk of SMA Type 1 by gene mutation analysis with bi-allelic SMN1 mutations and up to 2 copies of SMN2 (for presymptomatic participants)
- Age 2 weeks to younger than 5 months at time of dosing (for presymptomatic participants)
You will not qualify if you...
- Any suspected or confirmed active viral infection at screening baseline including HIV, Hepatitis B or C, or HTLV
- History of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry less than 95% saturation
- Ongoing or recent (within 3 months) immunosuppressive therapy such as corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, or rituximab
- Participation in a recent SMA treatment clinical trial that poses unnecessary risks for gene transfer
- Prior gene therapy, hematopoietic transplant, or solid organ transplant
- Severe scoliosis
- Known allergy or hypersensitivity to prednisolone, glucocorticosteroids, or their excipients
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 1 location
1
Hospital de Clínicas de Porto Alegre
Porto Alegre, Rio Grande do Sul, Brazil, 90035-903
Actively Recruiting
Research Team
J
Jenna Tress
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
SEQUENTIAL
Primary Purpose
TREATMENT
Number of Arms
4
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