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Study of Safety, Tolerability and Efficacy of GB221 in Infants With Spinal Muscular Atrophy Type 1

StatusRecruiting
PhasePhase 1/2
Started
View on ClinicalTrials.gov ↗

Record History

  1. Re-verified, no change to tracked fieldsv4
  2. Trial status: Not Yet Recruiting to Recruiting
    • Study sites: 0 to 1
    • Start date changed from estimate of Jan 2026 to Jan 6, 2026
    v3
  3. Start date: Oct 2025 to Jan 2026v2
  4. Study First Submitted
  • Stopped: an ongoing trial was terminated, suspended or withdrawn.
  • Notable: the trial’s plan changed such as a big move in its timeline or size, a new primary endpoint, sponsor or phase, or recruiting opened or resumed.
  • Minor: routine progress and housekeeping, such as sites added or enrollment closed.
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.
Trial Details
NCT Number NCT07070999
Lead Sponsor Gemma Biotherapeutics
Conditions
(from sponsor)		 Spinal Muscular Atrophy Type I
Related terms Spinal Muscular Atrophies of Childhood
Enrollment 22 participants
Start Date
Primary Completion (estimated)
Study Completion (estimated)
Updated on ClinicalTrials.gov