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.
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…
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