Gene Transfer Study in Patients With Late Onset Pompe Disease

NCTID	NCT04174105 (View at clinicaltrials.gov)
Description	This is a phase 1/2 open-label, ascending dose, multicenter clinical study to evaluate the safety and efficacy of AT845 in adult (aged ≥ 18 years) subjects, ambulatory or nonambulatory, with Late Onset Pompe Disease (LOPD). (Show More)
Development Status	Active
Indication	Pompe Disease (Late-onset)
Disease Ontology Term	DOID:2752
Compound Name	AT845
Compound Alias	Zocaglusagene nuzaparvovec
Sponsor	Astellas Gene Therapies
Funder Type	Industry
Recruitment Status	Active not recruiting
Enrollment Count	11
Results Posted	Not Available

Target Gene/Variant	GAA
Therapy Type	Gene transfer
Therapy Route	In-vivo
Mechanism of Action	Functional gene replacement
Route of Administration	Intravenous
Drug Product Type	Viral vector
Target Tissue/Cell
Delivery System	Viral transduction
Vector Type	AAV8
Editor Type	none
Dose 1	3E13 vg/kg
Dose 2	6E13 vg/kg
Dose 3	1E14 vg/kg
Dose 4
Dose 5

No.of Trial Sites	4
Locations	United States,United Kingdom

Has US IND	True
FDA Designations
Recent Updates	Anticipated PoC judgement timing: 2H/FY2025