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

Recruiting Phase 1 Phase 2 Interventional Study
Spinal Muscular Atrophy Type I
No Placebo Group Every participant receives an active treatment — no one gets a placebo. Prior Safety Data This treatment has already been tested in at least one earlier human trial.
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At a Glance
Age
2 Weeks – 12 Months
Sex
Any
Trial phase
Phase 1/2
Study type
Interventional
Purpose
Treatment
Participants needed
22 (estimated)
Sponsor
Gemma Biotherapeutics · Industry
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About This Trial
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. partic…
Trial Locations
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Eligibility Criteria
Inclusion Criteria: * Symptomatic Participants 1. 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 2. Participants must be 2 weeks to \< 12 months of age at the time of dosing with disease onset of d…
Contacts

Denise Reilly

267-718-7654

clinical_studies@gemmabiotx.com

CONTACT