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

NCTID	NCT04713475 (View at clinicaltrials.gov)
Description	PBGM01 is a gene therapy for GM1 gangliosidosis intended to deliver a functional copy of the GLB1 gene to the brain and peripheral tissues. This study will assess in a 2 part design the safety, tolerability and efficacy of PBGM01 in patients with early onset infantile (Type 1) and late onset infantile (Type 2a) GM1 gangliosidosis (Show More)
Development Status	Active
Indication	GM1 Gangliosidosis, Beta-Galactosidase-1 (GLB1) Deficiency
Disease Ontology Term	DOID:3322
Compound Name	PBGM01
Sponsor	Passage Bio, Inc.
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	26
Results Posted	Not Available

Target Gene/Variant	GLB1
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intracisterna magna
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAVhu68
Editor Type	none
Dose 1	3.3E10 GC/g brain weight
Dose 2	1.1E11 GC/g brain weight
Dose 3	2.2E11 GC/g brain weight
Dose 4	Undisclosed dose 4
Dose 5

No.of Trial Sites	9
Locations	Canada,Turkey,United States,Brazil,United Kingdom

Has US IND	True
FDA Designations
Recent Updates	Out-licensed development to Gemma Biotherapeutics, uncertain development status