19. Lysosomal storage disease Clinical trials / Disease details


Clinical trials : 854 Drugs : 716 - (DrugBank : 105) / Drug target genes : 70 - Drug target pathways : 191

  
9 trials found
No.TrialIDDate_
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PhaseCountries
1NCT04573023
(ClinicalTrials.gov)
February 14, 202217/9/2020A Phase ? Study of JR-141 in Patients With Mucopolysaccharidosis IIA Phase ? Study of JR-141 in Patients With Mucopolysaccharidosis IIMucopolysaccharidosis IIDrug: JR-141;Drug: Idursulfase;Drug: JR-141 or IdursulfaseJCR Pharmaceuticals Co., Ltd.NULLRecruitingN/AN/AMale50Phase 3United States
2JPRN-jRCT2031210126
31/05/202131/05/2021Post-marketing Clinical Study of IZCARGO in Patients with Mucopolysaccharidosis Type IIPost-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 TatsuyoshiNULLRecruitingNot applicableNot applicableBoth20Phase 4Japan
3NCT04348136
(ClinicalTrials.gov)
September 1, 201918/12/2019An Extension Study of JR-141 in Patients With Mucopolysaccharidosis IIAn Extension Study of JR-141-301 in Patients With Mucopolysaccharidosis IIMucopolysaccharidosis IIDrug: JR-141JCR Pharmaceuticals Co., Ltd.NULLActive, not recruitingN/AN/AMale27Phase 2/Phase 3Japan
4NCT03708965
(ClinicalTrials.gov)
January 1, 201927/9/2018An Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis IIAn Extension Study of JR-141-BR21 in Patients With Mucopolysaccharidosis IIMucopolysaccharidosis IIDrug: JR-141JCR Pharmaceuticals Co., Ltd.NULLActive, not recruitingN/AN/AMale18Phase 2Brazil
5NCT03568175
(ClinicalTrials.gov)
August 1, 20181/6/2018A Study of JR-141 in Patients With Mucopolysaccharidosis IIA Phase II/III Study of JR-141 in Patients With Mucopolysaccharidosis IIMucopolysaccharidosis IIDrug: JR-141JCR Pharmaceuticals Co., Ltd.NULLCompletedN/AN/AMale28Phase 2/Phase 3Japan
6NCT03359213
(ClinicalTrials.gov)
July 26, 201817/11/2017A Study of JR-141 in Patients With Mucopolysaccharidosis IIPhase II Study of JR-141 in Patients With Mucopolysaccharidosis IIMucopolysaccharidosis IIDrug: JR-141JCR Pharmaceuticals Co., Ltd.NULLCompletedN/AN/AMale18Phase 2Brazil
7NCT03128593
(ClinicalTrials.gov)
March 30, 201710/4/2017A Study of JR-141 in Patients With Mucopolysaccharidosis Type IIA Phase I/II Study of JR-141 in Patients With Mucopolysaccharidosis Type IIMucopolysaccharidosis IIDrug: JR-141JCR Pharmaceuticals Co., Ltd.NULLCompleted6 YearsN/AMale14Phase 1/Phase 2Japan
8EUCTR2020-003200-14-DE
(EUCTR)
15/06/2021A Phase III study of JR-141 in Hunter Syndrome patientsA 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.NULLNAFemale: no
Male: yes
50Phase 3France;United States;Brazil;Germany;United Kingdom
9EUCTR2020-003200-14-FR
(EUCTR)
17/06/2021A Phase III study of JR-141 in Hunter Syndrome patientsA 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.NULLNAFemale: no
Male: yes
50Phase 3United States;France;Brazil;Germany;United Kingdom