19. ライソゾーム病 Lysosomal storage disease Clinical trials / Disease details
臨床試験数 : 899 / 薬物数 : 684 - (DrugBank : 99) / 標的遺伝子数 : 51 - 標的パスウェイ数 : 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 |