19. Lysosomal storage disease Clinical trials / Disease details
Clinical trials : 854 / Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191
| 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 | NCT04573023 (ClinicalTrials.gov) | February 14, 2022 | 17/9/2020 | A Phase ? Study of JR-141 in Patients With Mucopolysaccharidosis II | A Phase ? Study of JR-141 in Patients With Mucopolysaccharidosis II | Mucopolysaccharidosis II | Drug: JR-141;Drug: Idursulfase;Drug: JR-141 or Idursulfase | JCR Pharmaceuticals Co., Ltd. | NULL | Recruiting | N/A | N/A | Male | 50 | Phase 3 | United States |
| 2 | JPRN-jRCT2031210126 | 31/05/2021 | 31/05/2021 | Post-marketing Clinical Study of IZCARGO in Patients with Mucopolysaccharidosis Type II | Post-marketing Clinical Study of IZCARGO in Patients with Mucopolysaccharidosis Type II | mucopolysaccharidosis type II (MPS II) | [Post-marketing clinical study drug] Name - Post-marketing clinical study drug (code): IZCARGO for I.V. infusion 10 mg (test drug code: JR-141) - Nonproprietary name International nonproprietary name (INN): pabinafusp alfa (r-INN List 82, WHO Drug Information, Vol. 33. No. 3, 2019) Japanese accepted names for pharmaceuticals (JAN) Registered number: 301-4-B3 Japanese name: pabinafusp alfa (genetical recombination) English name: Pabinafusp Alfa (Genetical Recombination) Dose and regimen: Subjects will receive 2.0 mg of pabinafusp alfa (genetical recombination) per kilogram of body weight once a week as an intravenous infusion. | Yamamoto Tatsuyoshi | NULL | Recruiting | Not applicable | Not applicable | Both | 20 | Phase 4 | Japan |
| 3 | NCT04348136 (ClinicalTrials.gov) | September 1, 2019 | 18/12/2019 | An Extension Study of JR-141 in Patients With Mucopolysaccharidosis II | An Extension Study of JR-141-301 in Patients With Mucopolysaccharidosis II | Mucopolysaccharidosis II | Drug: JR-141 | JCR Pharmaceuticals Co., Ltd. | NULL | Active, not recruiting | N/A | N/A | Male | 27 | Phase 2/Phase 3 | Japan |
| 4 | NCT03708965 (ClinicalTrials.gov) | January 1, 2019 | 27/9/2018 | An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II | An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis II | Mucopolysaccharidosis II | Drug: JR-141 | JCR Pharmaceuticals Co., Ltd. | NULL | Active, not recruiting | N/A | N/A | Male | 18 | Phase 2 | Brazil |
| 5 | NCT03568175 (ClinicalTrials.gov) | August 1, 2018 | 1/6/2018 | A Study of JR-141 in Patients With Mucopolysaccharidosis II | A Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis II | Mucopolysaccharidosis II | Drug: JR-141 | JCR Pharmaceuticals Co., Ltd. | NULL | Completed | N/A | N/A | Male | 28 | Phase 2/Phase 3 | Japan |
| 6 | NCT03359213 (ClinicalTrials.gov) | July 26, 2018 | 17/11/2017 | A Study of JR-141 in Patients With Mucopolysaccharidosis II | Phase II Study of JR-141 in Patients With Mucopolysaccharidosis II | Mucopolysaccharidosis II | Drug: JR-141 | JCR Pharmaceuticals Co., Ltd. | NULL | Completed | N/A | N/A | Male | 18 | Phase 2 | Brazil |
| 7 | NCT03128593 (ClinicalTrials.gov) | March 30, 2017 | 10/4/2017 | A Study of JR-141 in Patients With Mucopolysaccharidosis Type II | A Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type II | Mucopolysaccharidosis II | Drug: JR-141 | JCR Pharmaceuticals Co., Ltd. | NULL | Completed | 6 Years | N/A | Male | 14 | Phase 1/Phase 2 | Japan |
| 8 | EUCTR2020-003200-14-DE (EUCTR) | 15/06/2021 | A Phase III study of JR-141 in Hunter Syndrome patients | A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients. | Mucopolysaccharidosis type II MedDRA version: 20.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Pabinafusp alfa Product Code: JR-141 INN or Proposed INN: Pabinafusp alfa Other descriptive name: JR-141 | JCR Pharmaceuticals Co., Ltd. | NULL | NA | Female: no Male: yes | 50 | Phase 3 | France;United States;Brazil;Germany;United Kingdom | |||
| 9 | EUCTR2020-003200-14-FR (EUCTR) | 17/06/2021 | A Phase III study of JR-141 in Hunter Syndrome patients | A Phase III study of JR-141 in Mucopolysaccharidosis type II (Hunter Syndrome) patients. | Mucopolysaccharidosis type II MedDRA version: 20.1;Level: PT;Classification code 10056889;Term: Mucopolysaccharidosis II;System Organ Class: 10010331 - Congenital, familial and genetic disorders;Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16] | Product Name: Pabinafusp alfa Product Code: JR-141 INN or Proposed INN: Pabinafusp alfa Other descriptive name: JR-141 | JCR Pharmaceuticals Co., Ltd. | NULL | NA | Female: no Male: yes | 50 | Phase 3 | United States;France;Brazil;Germany;United Kingdom |