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RecruitingPhase 1Phase 2NCT07070999

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

Sponsor

Gemma Biotherapeutics

Enrollment

22 participants

Start Date

Jan 6, 2026

Study Type

INTERVENTIONAL

Conditions

Spinal Muscular Atrophy Type I

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.

Eligibility

Min Age: 2 WeeksMax Age: 12 Months

Inclusion Criteria6

  • 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 Criteria7

  • 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.
  • Prior history of gene therapy for any indication, hematopoietic transplant or solid organ transplant
  • Subjects with severe scoliosis
  • Known allergy or hypersensitivity to prednisolone or other glucocorticosteroids or their excipients.

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Interventions

BIOLOGICALGB221

GB221

Locations(1)

Hospital de Clínicas de Porto Alegre

Porto Alegre, Rio Grande do Sul, Brazil

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NCT07070999