- Trials with a EudraCT protocol (11)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
11 result(s) found for: N-acetylgalactosamine.
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EudraCT Number: 2004-000642-21 | Sponsor Protocol Number: ASB-03-06 | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A Multicenter, Multinational, Open-Label Extension Study of Recombinant Human N-acetylgalactosamine 4-sulfatase (rhASB) in Patients with Mucopolysaccharidosis VI | |||||||||||||
Medical condition: Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome) | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: IE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-003680-10 | Sponsor Protocol Number: AROAPOC3-3001 | Start Date*: 2022-01-13 | |||||||||||
Sponsor Name:Arrowhead Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 3 Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Familial Chylomicronemia Syndrome | |||||||||||||
Medical condition: Familial Chylomicronemia Syndrome | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: AT (Trial now transitioned) FR (Trial now transitioned) IE (Trial now transitioned) BE (Trial now transitioned) ES (Ongoing) HR (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2005-003512-30 | Sponsor Protocol Number: ASB-008 | Start Date*: 2007-04-05 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A Phase 4 Multi-center, Multi-national, Open-label, Randomized, Two Dose Level Study of Naglazyme (galsulfase) in Infants with Maroteaux-Lamy Syndrome (MPS VI) | |||||||||||||
Medical condition: Mucopolysaccharidosis Type VI (MPS VI; Maroteaux-Lamy Syndrome) | |||||||||||||
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Population Age: Newborns, Infants and toddlers, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Completed) PT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2022-001135-85 | Sponsor Protocol Number: AROAPOC3-2003 | Start Date*: 2022-09-06 | |||||||||||
Sponsor Name:Arrowhead Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 2 Open-Label Extension Study to Evaluate the Long-Term Safety and Efficacy of ARO-APOC3 in Adults with Dyslipidemia | |||||||||||||
Medical condition: Mixed Dyslipidemia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: HU (Trial now transitioned) PL (Trial now transitioned) NL (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2008-007365-23 | Sponsor Protocol Number: MOR-002 | Start Date*: 2009-03-12 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A Phase 1/2, Multicenter, Open-Label, Dose-Escalation Study to Evaluate the Safety, Tolerability, and Efficacy of BMN 110 in Subjects with Mucopolysaccharidosis IVA (Morquio Syndrome) | |||||||||||||
Medical condition: Mucopolysaccharidosis Type IV A | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-005217-14 | Sponsor Protocol Number: DCR-A1AT-202 | Start Date*: 2022-03-09 | |||||||||||
Sponsor Name:Dicerna Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 2 Open-Label Extension Study to Evaluate the Safety and Pharmacodynamics of Belcesiran in Patients with PiZZ Alpha-1 Antitrypsin Deficiency Associated Liver Disease | |||||||||||||
Medical condition: PiZZAlpha-1 Antitrypsin Deficiency Associated Liver Disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2021-000688-57 | Sponsor Protocol Number: AROAPOC3-2002 | Start Date*: 2021-11-10 | |||||||||||
Sponsor Name:Arrowhead Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Double-blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Mixed Dyslipidemia | |||||||||||||
Medical condition: Mixed Dyslipidemia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: HU (Completed) PL (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2020-003313-35 | Sponsor Protocol Number: DCR-A1AT-201 | Start Date*: 2022-01-18 | |||||||||||
Sponsor Name:Dicerna Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Phase 2, Randomized, Double-blind, Placebo-controlled Study Investigating Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Two Dose Levels of Belcesiran in Patients with Alpha-1 ... | |||||||||||||
Medical condition: PiZZAlpha-1 Antitrypsin Deficiency Associated Liver Disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) SE (Prematurely Ended) NL (Ongoing) PT (Prematurely Ended) BE (Completed) ES (Prematurely Ended) FR (Completed) AT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2021-000687-30 | Sponsor Protocol Number: AROAPOC3-2001 | Start Date*: 2021-10-04 | |||||||||||
Sponsor Name:Arrowhead Pharmaceuticals, Inc. | |||||||||||||
Full Title: A Double-Blind, Placebo-Controlled Phase 2b Study to Evaluate the Efficacy and Safety of ARO-APOC3 in Adults with Severe Hypertriglyceridemia | |||||||||||||
Medical condition: Severe Hypertriglyceridemia | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) HU (Completed) NL (Completed) PL (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2015-004702-42 | Sponsor Protocol Number: RG101-04 | Start Date*: 2016-01-15 | ||||||||||||||||
Sponsor Name:Regulus Therapeutics Inc. | ||||||||||||||||||
Full Title: A Multi-Center, Parallel Group, Open-Label, Phase 2 Study to Evaluate the Efficacy and Safety of a Single Subcutaneous Injection of RG-101 Combined with Oral GSK2878175 Taken Once Daily for 6, 9, o... | ||||||||||||||||||
Medical condition: Treatment Naïve, Genotype 1 and 3, Chronic Hepatitis C Patients | ||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | |||||||||||||||||
Trial protocol: HU (Completed) GR (Completed) ES (Completed) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2010-020199-45 | Sponsor Protocol Number: MOR-005 | Start Date*: 2011-06-08 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc. | |||||||||||||
Full Title: A Multicenter, Multinational, Extension Study to Evaluate the Long-Term Efficacy and Safety of BMN 110 in Patients with Mucopolysaccharidosis IVA (Morquio A Syndrome) | |||||||||||||
Medical condition: Mucopolysaccharidosis Type IVA | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) DE (Completed) IT (Completed) PT (Completed) DK (Completed) ES (Completed) | |||||||||||||
Trial results: View results |
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