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 | JPRN-jRCT2031220250 | 11/03/2020 | 07/08/2022 | Lon-term study of idursulfase beta in patients with mucopolysaccharidosis type II | Lon-term study of idursulfase beta in patients with mucopolysaccharidosis type II - CKK001 | Mucopolysaccharidosis type II;D016532 | [Investigative drug, dose, and administration method] - Investigative drug: Vial formulation filled with idursulfase beta for intracerebroventricular administration (Concentration 15 +/- 1.5 mg/mL, liquid volume 1.25 mL) - Dose and administration method: An implantable cerebrospinal fluid reservoir is attached to the subject's head, and idursulfase beta 30 mg is administered to the ventricles once every 4 weeks. | Yanagi Komei | NULL | Complete | >= 6month old | < 15age old | Male | 6 | Phase 3 | Japan |
| 2 | NCT02663024 (ClinicalTrials.gov) | December 2016 | 17/1/2016 | Study of Idursulfase-beta (GC1111) in Hunter Syndrome | Phase 2, Randomized, Double-blind, Active-controlled, Dose-ranging Study to Evaluate the Pharmacokinetics, Pharmacodynamics and Safety of Idursulfase-beta (GC1111) in Hunter Syndrome (Mucopolysaccharidosis II) Patients | Mucopolysaccharidosis II | Biological: idursulfase beta;Biological: idursulfase | Green Cross Corporation | NULL | Not yet recruiting | 5 Years | 35 Years | Male | 20 | Phase 2 | NULL |
| 3 | JPRN-JMA-IIA00350 | 29/07/2016 | 04/06/2018 | Phase I/II clinical trial of idursulfase beta for mucopolysaccharidosis type II | Phase I/II clinical trial of idursulfase beta for mucopolysaccharidosis type II | Patients at the age between 6 months and 15 years with severe type mucopolysaccharidosis type II intravenously administered isursulfase at least for 24 weeks | Intervention type:DRUG. Intervention1:BHP001, Dose form:INJECTION, Route of administration:OTHER. | Torayuki Okuyama | Haruo Shintaku | Completed | >=6 MONTHS | <15 YEARS | Male | 6 | Phase 1-2 | Japan |
| 4 | NCT01645189 (ClinicalTrials.gov) | July 2012 | 16/7/2012 | Safety and Efficacy of Hunterase | To Evaluate the Safety and Efficacy of Hunterase(Idursulfase-beta) in Hunter Syndrome Patients < 6 Years of Age Receiving Idursulfase Enzyme Replacement Therapy | Hunter Syndrome | Biological: Hunterase | Green Cross Corporation | NULL | Completed | N/A | 5 Years | Male | 6 | Phase 3 | Korea, Republic of |