RHGUS

( DrugBank: - / KEGG DRUG: - )

1 disease

ID	Disease name (Link within this page)	Number of trials
19	Lysosomal storage disease	12

19. Lysosomal storage disease

Clinical trials : 899 / Drugs : 684 - (DrugBank : 99) / Drug target genes : 51 - Drug target pathways : 182

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Showing 1 to 10 of 12 trials

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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	JPRN-jRCT2051190085	06/01/2020	19/12/2019	A long-term study of beta-glucuronidase enzyme replacement therapy for Sly disease	A long-term, open label, extension study to assess the efficacy and safety of beta-glucuronidase enz ...	Mucopolysaccharidosis type VII Mucopolysaccharidosis VII, Sly syndrome, beta-glucuronidase, vestroni ...	Investigational drug: Vestronidase alfa [Recombinant human beta-glucuronidase (rhGUS)], 4 mg/kg, IV, ...	Hamazaki Takashi	NULL	Recruiting	Not applicable	Not applicable	Both	4	Phase 3	Japan
2	EUCTR2015-000104-26-PT (EUCTR)	03/10/2016	03/05/2016	A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age receiving enzym ...	An Open-label Study of UX003 rhGUSEnzyme Replacement Therapy in MPS 7 Patients Less than 5 Years Old ...	Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Here ...	Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not availa ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	15	Phase 2	United States;Portugal;Spain
3	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 rhGUSEnzyme Replacement Therapy in Sub ...	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: pe ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	12	Phase 3	United States;Portugal;Mexico;Brazil
4	EUCTR2015-000104-26-ES (EUCTR)	05/04/2016	03/02/2016	A safety, tolerability and efficacy study in MPS 7 patients less than 5 years of age receiving enzym ...	An Open-label Study of UX003 rhGUSEnzyme Replacement Therapy in MPS 7 Patients Less than 5 Years Old ...	Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Here ...	Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not availa ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	15	Phase 2	Portugal;United States;Spain
5	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 rhGUSEnzyme Replacement Therapy in Sub ...	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
6	NCT02418455 (ClinicalTrials.gov)	July 21, 2015	12/4/2015	Study of UX003 Recombinant Human Beta-Glucuronidase (rhGUS) Enzyme Replacement Treatment in Mucopoly ...	An Open-label Study of UX003 rhGUSEnzyme Replacement Therapy in MPS 7 Patients Less Than 5 Years Old ...	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
7	NCT02230566 (ClinicalTrials.gov)	December 2014	22/8/2014	A Phase 3 Study of UX003 Recombinant Human Betaglucuronidase (rhGUS) Enzyme Replacement Therapy in P ...	A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy ...	MPS 7;Sly Syndrome;Mucopolysaccharidosis;MPS VII	Drug: UX003;Other: Placebo	Ultragenyx Pharmaceutical Inc	NULL	Completed	5 Years	35 Years	All	12	Phase 3	United States
8	EUCTR2013-001152-35-ES (EUCTR)	21/08/2014	04/07/2014	A safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous in ...	An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUSEnzyme Replaceme ...	Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Here ...	Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not availa ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	5	Phase 1;Phase 2	Spain;United Kingdom
9	EUCTR2013-001152-35-GB (EUCTR)	02/08/2013	25/06/2013	A safety and efficacy study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous in ...	An Open-Label Phase 1/2 Study to Assess the Safety, Efficacy and Dose of UX003 rhGUSEnzyme Replaceme ...	Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Here ...	Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not availa ...	Ultragenyx Pharmaceutical Inc.	NULL	Not Recruiting			Female: yes Male: yes	5	Phase 1;Phase 2	Spain;United Kingdom
10	EUCTR2014-005638-71-Outside-EU/EEA (EUCTR)		22/12/2014	An efficacy and safety study in MPS 7 patients receiving enzyme (UX003) replacement by intravenous i ...	A Randomized, Placebo-Controlled, Blind-Start, Single-Crossover Phase 3 Study to Assess the Efficacy ...	Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome);Therapeutic area: Diseases [C] - Congenital, Here ...	Product Name: Recombinant human beta-glucuronidase Product Code: UX003 INN or Proposed INN: Not availa ...	Ultragenyx Pharmaceutical Inc.	NULL	NA			Female: yes Male: yes	12	Phase 3	United States

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