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

NCT07070999 · Status: RECRUITING · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 22

Last updated 2026-03-02

No results posted yet for this study

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.

Conditions

  • Spinal Muscular Atrophy Type I

Interventions

BIOLOGICAL

GB221

GB221

Sponsors & Collaborators

  • Gemma Biotherapeutics

    lead INDUSTRY

Principal Investigators

  • May Orfali, MD · Gemma Biotherapeutics

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SEQUENTIAL

Eligibility

Min Age
2 Weeks
Max Age
12 Months
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2026-01-06
Primary Completion
2028-12-31
Completion
2029-04-30

Countries

  • Brazil

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT07070999 on ClinicalTrials.gov