Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1 (NCT07070999) | Clinical Trial Compass
RecruitingPhase 1/2
Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1
Brazil22 participantsStarted 2026-01-06
Plain-language summary
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.
Who can participate
Age range2 Weeks – 12 Months
SexALL
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Inclusion criteria
✓. 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.
✓. 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 criteria
✕. 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.
✕. Ongoing immunosuppressive therapy or immunosuppressive therapy within 3 months of starting the trial (e.g. corticosteroids, cyclosporine, tacrolimus, methotrexate, cyclophosphamide, intravenous immunoglobulin, rituximab)
What they're measuring
1
Number of participants with treatment-related adverse events (AEs) and serious adverse events (SAEs) at Grade 3 or higher as characterized by CTCAEv5.0
Timeframe: Up to 18 months across multiple visits
2
Number of Participants with Clinically Significant Changes in Physical Functions
Timeframe: Up to 18 months across multiple visits
3
Number of Participants with Clinically Significant Changes in Neurological Functions
Timeframe: Up to 18 months across multiple visits
4
Number of Participants with Clinically Significant Changes in Vital signs
Timeframe: Up to 18 months across multiple visits
5
Change in electrocardiogram results
Timeframe: Up to 18 months across multiple visits
6
Change in serum cardiac troponin I levels
Timeframe: Up to 18 months across multiple visits
7
Number of Participants with Clinically Significant Laboratory Abnormalities as Measured Using Hematology, Chemistry and Coagulation Tests