NCT04273269 - A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis | Biotech Hunter

Enrollment

Participants

Timeline

Start Date

May 11, 2021

Primary Completion Date

May 22, 2023

Study Completion Date

May 22, 2023

Conditions

GM1 Gangliosidosis

Interventions

GENETIC

LYS-GM101

LYS-GM101 is an adeno-associated viral vector serotype rh.10 (AAVrh.10) carrying the human β-galactosidase gene, formulated as a suspension for injection

Trial Locations (3)

75012

Hôpital Armand-Trousseau, Centre de Référence des Maladies Lysosomales (CRML), Service de Neuropédiatrie, Paris

92868

Children's Hospital of Orange County (CHOC), Orange

M13 9WL

Manchester University NHS Foundation Trust, Manchester