NCT07070999RecruitingPhase 1/2
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
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
Source: ClinicalTrials.gov · Last updated 29 Jun 2026
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 22 (estimated)
- First submitted
- 30 May 2025
- Start date
- 6 Jan 2026
- Primary completion date
- Dec 2028
- Lead sponsor
- Gemma Biotherapeutics
- Diseases
- Spinal muscular atrophy
- NCT number
- NCT07070999
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 22
- From 6 Jan 2026 to the primary completion date, Dec 2028
- 1 site in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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
Other illness
- 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.
Other
- 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
Who the study is looking for
- 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.
Age in the registry: 2 weeks to 12 months · Sex: All · Does not accept healthy volunteers
Sites (1)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Hospital de Clinicas de Porto Alegre Coordinator of this site? Confirm its status | Porto Alegre | Brazil | Recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.