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

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

Plain Language Summary

Simplified for easier understanding

This clinical trial is studying a biological treatment called GB221 for people with spinal muscular atrophy type i. The study is currently recruiting participants at 1 location.

This summary was AI-generated to explain the trial in plain language. It is not medical advice. Always discuss eligibility with your doctor before enrolling in a clinical trial.

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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