Recombinant human beta-glucuronidase (rhGUS) ( DrugBank: Recombinant human beta-glucuronidase )


1 disease
告示番号疾患名(ページ内リンク)臨床試験数
19ライソゾーム病4

19. ライソゾーム病


臨床試験数 : 854 薬物数 : 716 - (DrugBank : 105) / 標的遺伝子数 : 70 - 標的パスウェイ数 : 191
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PhaseCountries
1JPRN-jRCT2051190085
06/01/202019/12/2019A long-term study of beta-glucuronidase enzyme replacement therapy for Sly diseaseA long-term, open label, extension study to assess the efficacy and safety of beta-glucuronidase enzyme replacement therapy in Japanese patients with mucopolysaccharidosis type VII, Sly disease Mucopolysaccharidosis type VII
Mucopolysaccharidosis VII, Sly syndrome, beta-glucuronidase, vestronidase alfa;D016538
Investigational drug: Vestronidase alfa [Recombinant human beta-glucuronidase (rhGUS)], 4 mg/kg, IV, QOWHamazaki TakashiNULLRecruitingNot applicableNot applicableBoth3Phase 3Japan
2EUCTR2015-001875-32-PT
(EUCTR)
18/04/201619/01/2016A long-term safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injectionA Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7 Mucopolysaccharidosis type 7 ( MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Product Name: Recombinant human beta-glucuronidase (rhGUS)
Product Code: UX003
INN or Proposed INN: pending
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
Ultragenyx Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
12 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (Phase 3): yes Therapeutic use (Phase 4): noUnited States;Portugal;Mexico;Brazil
3NCT02432144
(ClinicalTrials.gov)
November 10, 201522/4/2015A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Therapy in Subjects With Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7)A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects With MPS 7Sly Syndrome;MPS VII;Mucopolysaccharidosis;Mucopolysaccharidosis VIIDrug: UX003Ultragenyx Pharmaceutical IncNULLCompleted5 YearsN/AAll12Phase 3United States;Brazil;Mexico;Portugal
4NCT02418455
(ClinicalTrials.gov)
July 21, 201512/4/2015Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopolysaccharidosis Type 7, Sly Syndrome (MPS 7) Patients Less Than 5 Years of AgeAn Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years OldSly Syndrome;MPS VII;Mucopolysaccharidosis;Mucopolysaccharidosis VIIDrug: UX003Ultragenyx Pharmaceutical IncNULLCompletedN/A5 YearsAll8Phase 2United States;Portugal;Spain