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Clinical trial

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

NCT07070999 is registered in ClinicalTrials.gov with Gemma Biotherapeutics as lead sponsor.

NCT NCT07070999
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Registry record

As filed in ClinicalTrials.gov.

Title
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
Lead sponsor
Gemma Biotherapeutics
Phase
Phase 1/2
Overall status
Recruiting
Start date
January 6, 2026
Completion date
April 30, 2029
Enrollment
22 (estimated)
Sites
1

Conditions

As the registry lists them; a condition with its own page is linked.

spinal muscular atrophy type i