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

  
10 trials found
No.TrialIDDate_
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PhaseCountries
1NCT05594992
(ClinicalTrials.gov)
February 22, 20236/10/2022An Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Male SubjectsAn Extension Study of JR-141 to Evaluate the Long-term Safety and Efficacy in Mucopolysaccharidosis Type II (Hunter Syndrome) Male SubjectsMucopolysaccharidosis IIDrug: JR-141JCR Pharmaceuticals Co., Ltd.NULLEnrolling by invitationN/AN/AMale80Phase 3United States
2EUCTR2020-003200-14-DE
(EUCTR)
16/05/202215/06/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
50Phase 3France;United States;Brazil;Germany;United Kingdom
3EUCTR2020-003200-14-FR
(EUCTR)
04/05/202217/06/2021A 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.NULLAuthorised-recruitment may be ongoing or finishedFemale: no
Male: yes
50Phase 3United States;France;Brazil;Germany;United Kingdom
4NCT04573023
(ClinicalTrials.gov)
February 14, 202217/9/2020A Phase III Study of JR-141 in Patients With Mucopolysaccharidosis IIA Phase III Study of JR-141 in Patients With Mucopolysaccharidosis IIMucopolysaccharidosis IIDrug: JR-141;Drug: Idursulfase;Drug: JR-141 or IdursulfaseJCR Pharmaceuticals Co., Ltd.NULLRecruitingN/AN/AMale80Phase 3United States;Brazil;France;Germany;Spain
5JPRN-jRCT2031210126
27/07/202131/05/2021Post-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
6NCT04348136
(ClinicalTrials.gov)
September 1, 201918/12/2019An Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type IIAn Extension Study of JR-141 in Patients With Mucopolysaccharidosis Type IIMucopolysaccharidosis IIDrug: JR-141JCR Pharmaceuticals Co., Ltd.NULLActive, not recruitingN/AN/AMale27Phase 2/Phase 3Japan
7NCT03708965
(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 recruiting0 YearsN/AMale19Phase 2Brazil
8NCT03568175
(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
9NCT03359213
(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.NULLCompleted0 YearsN/AMale18Phase 2Brazil
10NCT03128593
(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