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

3 trials found

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No.	TrialID	Date_ enrollment	Date_ registration	Public_title	Scientific_title	Condition	Intervention	Primary_ sponsor	Secondary_ sponsor	Recruitment_ Status	Inclusion_ agemin	Inclusion_ agemax	Inclusion_ gender	Target_ size	Phase	Countries
1	EUCTR2015-001875-32-PT (EUCTR)	18/04/2016	19/01/2016	A long-term safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intr ...	A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Su ...	Mucopolysaccharidosis type 7 ( MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Her ...	Product Name: Recombinant human beta-glucuronidase (rhGUS) Product Code: UX003 INN or Proposed INN: pen ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	12	Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): no Therapeutic confirmatory - (P ...	United States;Portugal;Mexico;Brazil
2	NCT02432144 (ClinicalTrials.gov)	November 10, 2015	22/4/2015	A Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS)Enzyme Replacement Therapy in Subjects ...	A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Su ...	Sly Syndrome;MPS VII;Mucopolysaccharidosis;Mucopolysaccharidosis VII	Drug: UX003	Ultragenyx Pharmaceutical Inc	NULL	Completed	5 Years	N/A	All	12	Phase 3	United States;Brazil;Mexico;Portugal
3	NCT02418455 (ClinicalTrials.gov)	July 21, 2015	12/4/2015	Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS)Enzyme Replacement Treatment in Mucopolys ...	An Open-label Study of UX003 rhGUS Enzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Ol ...	Sly Syndrome;MPS VII;Mucopolysaccharidosis;Mucopolysaccharidosis VII	Drug: UX003	Ultragenyx Pharmaceutical Inc	NULL	Completed	N/A	5 Years	All	8	Phase 2	United States;Portugal;Spain

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