LYS-GM101 ( DrugBank: - )

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1 disease

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	2

19. Lysosomal storage disease

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Clinical trials : 899 /  Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	NCT04273269 (ClinicalTrials.gov)	May 11, 2021	21/1/2020	A Safety and Efficacy Study of LYS-GM101 Gene Therapy in Patients With GM1 Gangliosidosis	An Open-Label Adaptive-Design Study of Intracisternal Adenoassociated Viral Vector Serotype rh.10 Carrying the Human ß-Galactosidase cDNA for Treatment of GM1 Gangliosidosis	GM1 Gangliosidosis	Genetic: LYS-GM101	LYSOGENE	NULL	Active, not recruiting	N/A	3 Years	All	16	Phase 1/Phase 2	United States;France;United Kingdom
2	EUCTR2019-004949-32-FR (EUCTR)		09/03/2020	LYS-GM101 gene therapy trial in patients with GM1 gangliosidosis	An open-label adaptive-design study of intracisternal administration of adeno-associated viral vector serotype rh.10 carrying the human ß-galactosidase cDNA for the treatment of GM1 gangliosidosis	GM1 gangliosidosis MedDRA version: 20.0;Level: SOC;Classification code 10010331;Term: Congenital, familial and genetic disorders;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]	Product Name: LYS-GM101 Product Code: LYS-GM101 INN or Proposed INN: adeno-associated virus (AAV) serotype rh.10 expressing human betagalactosidase (AAVrh.10-CAG-ßgal) Other descriptive name: Adeno-associated virus serotype rh.10 expressing beta-galactosidase	Lysogene	NULL	NA			Female: yes Male: yes	18	Phase 1;Phase 2	United States;France;United Kingdom