Iduronate 2-sulfatase ( DrugBank: Iduronate 2-sulfatase )


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2

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
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PhaseCountries
1EUCTR2018-000192-33-GB
(EUCTR)
17/08/201809/04/2018A phase 1/2 study to evaluate the safety and tolerability of SB-913, a rAAV2/6-based gene therapy in patients with Mucopolysaccharidosis II (MPS II)A Phase 1/2, Multicenter, Open-label, Single-dose, Dose-ranging Study to Assess the Safety and Tolerability of SB-913, a rAAV2/6-based Gene Transfer in Subjects with Mucopolysaccharidosis II (MPS II) Mucopolysaccharidosis type II (MPS 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] - Nutritional and Metabolic Diseases [C18]
Product Name: rAAV2/6 Left ZFN Vector
Product Code: SB-47171
INN or Proposed INN: Not yet assigned
Other descriptive name: Adeno-associated virus serotype 2/6 encoding Left side-zinc finger nuclease (ZFN1), SB-A6P-ZLEFT Vector
Product Name: rAAV2/6 Right ZFN Vector
Product Code: SB-47898
INN or Proposed INN: Not yet assigned
Other descriptive name: Adeno-associated virus serotype 2/6 encoding Right side-zinc finger nuclease (ZFN2), SB-A6P-ZRIGHT Vector
Product Name: rAAV 2/6 hIDS DONOR
Product Code: hIDS DONOR
INN or Proposed INN: Not yet assigned
Other descriptive name: Adeno-associated virus serotype 2/6 encoding Human Iduronate 2-sulfatase (hIDS) gene donor vector, SB-A6P-HNT Donor Vector
Sangamo Therapeutics, Inc.NULLAuthorised-recruitment may be ongoing or finished Female: no
Male: yes
32 Human pharmacology (Phase 1): yes Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;United Kingdom
2NCT02262338
(ClinicalTrials.gov)
April 20152/10/2014Safety and Dose Ranging Study of Insulin Receptor MoAb-IDS Fusion Protein in Patients With Hunter SyndromeA Phase 1 Safety and Dose-Finding Study of a Human Insulin Receptor Monoclonal Antibody-Human Iduronate 2-Sulfatase (IDS) Fusion Protein, AGT-182 in Adult Patients With Mucopolysaccharidosis II (MPS II, Hunter Syndrome)Mucopolysaccharidosis IIDrug: AGT-182ArmaGen, IncNULLCompleted18 YearsN/AMale6Phase 1United States;Germany;Philippines;Netherlands