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.
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
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)
Participation in a recent SMA treatment clinical trial that, in the opinion of the Investigator, creates unnecessary risks for gene transfer.
Sequential
GB221
Biological/VaccineGB221
Number of participants with treatment-related adverse events (AEs) and serious adverse events (SAEs) at Grade 3 or higher as characterized by CTCAEv5.0
Assess the number of treatment-related AEs and SAEs as characterized by CTCAEv5.0
Number of Participants with Clinically Significant Changes in Physical Functions
Assess the number of participants with clinically significant changes in physical functions.
Number of Participants with Clinically Significant Changes in Neurological Functions
Assess the number of participants with clinically significant changes in neurological functions.
Number of Participants with Clinically Significant Changes in Vital signs
Assess the number of participants with clinically significant changes in vital signs.
Assess the number of participants who experience permanent ventilation or death
Percentage of infants with improvement in the motor milestones categories in Section 2 of the Hammersmith Infant Neurological Examination (HINE), with the exclusion of voluntary grasp.
* improvement in at least one category, i.e., an increase in the score for head control, rolling, sitting, crawling, standing, or walking of ≥1 point, * an increase in the score for kicking of ≥2 points, or achievement of the maximal score for kicking.
Change from baseline in mean Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders (CHOP-INTEND) Score.
Assess 16 types of muscle movements, each given a score from zero (the person can't complete the movement) to 4 (the person can complete the movement on their own, without assistance) to produce a score of 0 to 64.
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