GNR-055 ( 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	NCT05208281 (ClinicalTrials.gov)	November 30, 2021	14/10/2021	A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II	Multicenter, Open-Label, Multi-cohort Study to Evaluate Safety, Pharmacokinetics, Pharmacodynamics, and Efficacy of Drug Product GNR 055 (JSC GENERIUM, Russia) in Patients With Mucopolysaccharidosis Type II	Mucopolysaccharidosis Type II;Metabolic Diseases	Drug: GNR-055 1.0-2.0-3.0 mg/kg;Drug: GNR-055 2.0 mg/kg;Drug: GNR-055 3.0 mg/kg	AO GENERIUM	NULL	Recruiting	N/A	N/A	Male	32	Phase 2/Phase 3	Russian Federation
2	NCT04539340 (ClinicalTrials.gov)	February 28, 2020	28/8/2020	A Multi-cohort Study of the Tolerance, Safety, and Pharmacokinetics of GNR-055 in Healthy Volunteers	An Open-label Multi-cohort Dose-escalation Study to Evaluate the Tolerance, Safety, and Pharmacokinetics of GNR-055 (GENERIUM JSC, Russia) in Healthy Volunteers With a Single Intravenous Administration	Mucopolysaccharidosis Type II;Metabolic Diseases	Biological: GNR-055	AO GENERIUM	NULL	Completed	18 Years	50 Years	Male	20	Phase 1	Russian Federation