RHGUS ( DrugBank: - )


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12

19. ライソゾーム病


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.TrialIDDate_
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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 applicableBoth4Phase 3Japan
2EUCTR2015-000104-26-PT
(EUCTR)
03/10/201603/05/2016A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age receiving enzyme (UX003) replacement by intravenous injectionAn Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 Years Old 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
Product Code: UX003
INN or Proposed INN: Not available
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
Ultragenyx Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
15 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Portugal;Spain
3EUCTR2015-001875-32-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
4EUCTR2015-000104-26-ES
(EUCTR)
05/04/201603/02/2016A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age receiving enzyme (UX003) replacement by intravenous injectionAn Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 Years Old 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
Product Code: UX003
INN or Proposed INN: Not available
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
Ultragenyx Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
15 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noPortugal;United States;Spain
5NCT02432144
(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
6NCT02418455
(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
7NCT02230566
(ClinicalTrials.gov)
December 201422/8/2014A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in Patients With Mucopolysaccharidosis Type 7 (MPS 7)A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients With MPS 7MPS 7;Sly Syndrome;Mucopolysaccharidosis;MPS VIIDrug: UX003;Other: PlaceboUltragenyx Pharmaceutical IncNULLCompleted5 Years35 YearsAll12Phase 3United States
8EUCTR2013-001152-35-ES
(EUCTR)
21/08/201404/07/2014A safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injectionAn Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients 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
Product Code: UX003
INN or Proposed INN: Not available
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
Ultragenyx Pharmaceutical Inc.NULLNot RecruitingFemale: yes
Male: yes
5Phase 1;Phase 2Spain;United Kingdom
9EUCTR2013-001152-35-GB
(EUCTR)
02/08/201325/06/2013A safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injectionAn Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUS Enzyme Replacement Therapy in Patients 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
Product Code: UX003
INN or Proposed INN: Not available
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
Ultragenyx Pharmaceutical Inc.NULLNot Recruiting Female: yes
Male: yes
5 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noSpain;United Kingdom
10EUCTR2015-000104-26-Outside-EU/EEA
(EUCTR)
23/07/2015A safety and efficacy study in young MPS 7 patients (less than 5 years old) receiving enzyme (UX003) replacement by intravenous injectionAn Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less than 5 years old Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]Product Name: Recombinant human betaglucuronidase
Product Code: UX003
INN or Proposed INN: pending
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
Ultragenyx Pharmaceutical Inc.NULLNAFemale: yes
Male: yes
7United States
11EUCTR2015-001875-32-Outside-EU/EEA
(EUCTR)
12/11/2015A 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
Product Code: UX003
INN or Proposed INN: pending
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
Ultragenyx Pharmaceutical Inc.NULLNAFemale: yes
Male: yes
12United States
12EUCTR2014-005638-71-Outside-EU/EEA
(EUCTR)
22/12/2014An efficacy and safety study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous injectionA Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy and Safety of UX003 rhGUS Enzyme Replacement Therapy in Patients 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
Product Code: UX003
INN or Proposed INN: Not available
Other descriptive name: RECOMBINANT HUMAN BETA GLUCURONIDASE; RHGUS
Ultragenyx Pharmaceutical Inc.NULLNAFemale: yes
Male: yes
12Phase 3United States