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Clinical trial

Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis

NCT04713475 is registered in ClinicalTrials.gov with Gemma Biotherapeutics as lead sponsor.

NCT NCT04713475
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Registry record

As filed in ClinicalTrials.gov.

Title
Phase 1/2 Open-Label, Multicenter Study to Assess the Safety, Tolerability and Efficacy of a Single Dose of PBGM01 Delivered Into the Cisterna Magna of Pediatric Type 1 (Early Onset) and Type 2a (Late Onset) Infantile GM1 Gangliosidosis
Lead sponsor
Gemma Biotherapeutics
Phase
Phase 1/2
Overall status
Active, not recruiting
Start date
March 17, 2021
Completion date
February 28, 2029
Enrollment
26 (estimated)
Sites
7

Conditions

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

beta-galactosidase-1 (glb1) deficiency, gm1 gangliosidosis, gm1 gangliosidosis, type 2, gm1 gangliosidosis, type i