Find Spinal Muscular Atrophy Type 3 Clinical Trials Near You
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
Status: Recruiting
Location: See location...
Intervention Type: Biological
Study Type: Interventional
Study Phase: Phase 1/Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 14 days
Maximum Age: 1
Healthy Volunteers: f
View:
• 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.
Locations
Other Locations
Brazil
Hospital de Clínicas de Porto Alegre
RECRUITING
Porto Alegre
Contact Information
Primary
Jenna Tress
clinical_studies@gemmabiotx.com
445-895-3356
Time Frame
Start Date:2026-01-06
Estimated Completion Date:2029-04
Participants
Target number of participants:22
Treatments
Experimental: Cohort 1A, safety and exploratory efficacy of a single dose in symptomatic participants
Symptomatic participants with SMA Type 1 (up to 3 copies of SMN2), who are either treatment naïve or receiving risdiplam, with onset of disease during the first 6 months of life, aged from 2 weeks to younger than 12 months at the time of dosing.
Experimental: Cohort 1B, expansion phase for confirmatory testing in symptomatic participants
Symptomatic participants with SMA Type 1 (up to 3 copies of SMN2), who are either treatment naïve or receiving risdiplam, with onset of disease during the first 6 months of life, aged from 2 weeks to younger than 12 months at the time of dosing.
Experimental: Cohort 2A, safety and exploratory efficacy of a single dose in presymptomatic participants
Presymptomatic participants (treatment naïve or receiving risdiplam) at risk of developing SMA Type 1 (up to 2 copies of SMN2), aged from 2 weeks to younger than 5 months (\< 150 days) at the time of dosing.
Experimental: Cohort 2B, expansion phase for confirmatory testing in presymptomatic participants
Presymptomatic participants (treatment naïve or receiving risdiplam) at risk of developing SMA Type 1 (up to 2 copies of SMN2), aged from 2 weeks to younger than 5 months (\< 150 days) at the time of dosing.