Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis | ClinicalTrials HQ

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Trial	Conditions	Status	Phase	Updated	More info	NCT ID
Study of Safety, Tolerability and Efficacy of PBGM01 in Pediatric Participants With GM1 Gangliosidosis	Brain Diseases, Metabolic, Brain Diseases, Metabolic, Inborn, Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Disease Attributes, GM1 gangliosidosis, Gangliosidoses, Gangliosidosis, Generalized GM1, Type 1, Lipid Metabolism Disorders, Lipid Metabolism, Inborn Errors, Lysosomal Storage Diseases, Nervous System, Nutritional and Metabolic Diseases, Pathologic Processes, Pathological Conditions, Signs and Symptoms, Rare Diseases, beta-Galactosidase Deficiency, brain disorder, central nervous system disorder, hereditary disease, inborn errors of metabolism, lysosomal lipid storage disorder, lysosomal storage disease, metabolic disease, nervous system disorder, sphingolipidosis	Active, not recruiting	Phase 1 / Phase 2	2026-06-30		NCT04713475

1 results · Page 1 of 1

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Organizations	Linked trials
Gemma Biotherapeutics	2

7 results · Page 1 of 1

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Research sites	City	Country	Linked trials
Benioff Children's Hospital	Oakland	United States	1
Children's Hospital at St. Peter's University Hospital	New Brunswick	United States	1
Gazi University	Ankara	Turkey (Türkiye)	15
Great Ormond Street Hospital	London	United Kingdom	15
Hospital de Clínicas de Porto Alegre (HCPA)	Porto Alegre	Brazil	1
The Children's Hospital of Philadelphia	Philadelphia	United States	62
University of Minnesota	Minneapolis	United States	325