Tests GB221 gene therapy in babies with spinal muscular atrophy type 1
Tests GB221 gene therapy in babies with spinal muscular atrophy type 1.
Plain-language title generated from the registry record
Registry title: Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
Key facts
- Where
- Porto Alegre, BrazilSee the site
- Phase
- Phase 1/2
Where in Brazil
- Hospital de Clinicas de Porto AlegrePorto Alegre, BrazilTaking people
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Gemma Biotherapeutics
- Trial code
- NCT07070999
- Record updated
- 29 June 2026
- Original description
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:Read full original descriptionShow less
A Phase 1-2, Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of GB221 Delivered Into the Cisterna Magna of Pediatric Participants From 2 Weeks to Younger Than 12 Months of Age With Spinal Muscular Atrophy Type 1
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.
Inclusion: Symptomatic Participants; Diagnosis of SMA Type 1 based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and up to 3 copies of SMN2; Participants must be 2 weeks to < 12 months of age at the time of dosing with disease onset of during the first 6 months of life; Presymptomatic Participants; At risk of SMA Type 1 based on gene mutation analysis with bi-allelic SMN1 mutations (deletion or point mutations) and up to 2 copies of SMN2; Participants must be 2 weeks to < 5 months (< 150 days) of age at the time of dosing.
Exclusion: Ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial (e.g. corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab); Prior history of gene therapy for any indication, hematopoietic transplant or solid organ transplant; Any suspected or confirmed active viral infection at screening baseline (including HIV, Hepatitis B or C, or human T Cell lymphotropic viruses [HTLV]); History of invasive ventilatory support (tracheotomy with positive pressure) or pulse oximetry <95% saturation; Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients; Participation in a recent SMA treatment clinical trial that, in the opinion of the Investigator, creates unnecessary risks for gene transfer; Subjects with severe scoliosis.