- Trials with a EudraCT protocol (9)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
9 result(s) found for: Enzyme substrate.
Displaying page 1 of 1.
EudraCT Number: 2020-005032-30 | Sponsor Protocol Number: FLT201-01 | Start Date*: 2023-03-13 | ||||||||||||||||
Sponsor Name:Freeline Therapeutics Limited | ||||||||||||||||||
Full Title: A Phase 1/2, open-label, safety, tolerability and efficacy study of FLT201 in adult patients with Gaucher disease Type 1 (Galileo-1) | ||||||||||||||||||
Medical condition: Type 1 Gaucher Disease | ||||||||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | |||||||||||||||||
Trial protocol: ES (Ongoing) | ||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2015-001875-32 | Sponsor Protocol Number: UX003-CL202 | Start Date*: 2015-11-12 |
Sponsor Name:Ultragenyx Pharmaceutical Inc. | ||
Full Title: A Long-Term Open-Label Treatment and Extension Study of UX003 rhGUS Enzyme Replacement Therapy in Subjects with MPS 7 | ||
Medical condition: Mucopolysaccharidosis type 7 (MPS 7, Sly syndrome) | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18, Adults | Gender: Male, Female | |
Trial protocol: Outside EU/EEA PT (Completed) | ||
Trial results: View results |
EudraCT Number: 2018-000755-40 | Sponsor Protocol Number: ATB200-03 | Start Date*: 2019-04-08 | |||||||||||
Sponsor Name:Amicus Therapeutics, Inc. | |||||||||||||
Full Title: A Phase 3 Double-blind Randomized Study to Assess the Efficacy and Safety of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late Onset Pompe Disease Compared With Algluc... | |||||||||||||
Medical condition: Adult Subjects With Late Onset Pompe Disease (LOPD) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) DK (Completed) GB (GB - no longer in EU/EEA) SE (Completed) HU (Completed) BE (Completed) SK (Completed) ES (Completed) NL (Completed) BG (Prematurely Ended) SI (Completed) GR (Completed) AT (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-000954-67 | Sponsor Protocol Number: ATB200-07 | Start Date*: 2020-04-08 | |||||||||||
Sponsor Name:Amicus Therapeutics, Inc. | |||||||||||||
Full Title: A Phase 3 Open-label Extension Study to Assess the Long-term Safety and Efficacy of Intravenous ATB200 Co-administered With Oral AT2221 in Adult Subjects With Late-onset Pompe Disease | |||||||||||||
Medical condition: Adult Subjects With Late Onset Pompe Disease (LOPD) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) ES (Ongoing) DE (Completed) DK (Trial now transitioned) FR (Trial now transitioned) BE (Trial now transitioned) GR (Trial now transitioned) SI (Trial now transitioned) AT (Completed) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2007-000498-42 | Sponsor Protocol Number: PB-06-001 | Start Date*: 2008-05-08 | |||||||||||
Sponsor Name:Protalix Biotherapeutics | |||||||||||||
Full Title: A Phase III Multicenter, Randomized, Double-Blind Trial to Assess the Safety and Efficacy of Two Parallel Dose Groups of Plant Cell Expressed Recombinant Human Glucocerebrosidase (prGCD) in Patient... | |||||||||||||
Medical condition: Gaucher disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) ES (Completed) IT (Completed) Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2005-004384-33 | Sponsor Protocol Number: AA1565522 (FAB-CL-203) | Start Date*: 2005-12-19 | |||||||||||
Sponsor Name:Amicus Therapeutics | |||||||||||||
Full Title: A Phase 2, Open-Label, Single Dose Level, 24-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Patients with Fabry Disease | |||||||||||||
Medical condition: Fabry Disease | |||||||||||||
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Population Age: Adults | Gender: Male | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2006-000181-36 | Sponsor Protocol Number: FAB-CL-204 | Start Date*: 2006-03-17 | |||||||||||
Sponsor Name:Amicus Therapeutics, Inc. | |||||||||||||
Full Title: A Phase 2, Open-Label, Multiple Dose Level, 12-Week Study to Evaluate the Safety, Tolerability, and Pharmacodynamics of AT1001 in Female Patients with Fabry Disease | |||||||||||||
Medical condition: Fabry disease | |||||||||||||
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Population Age: Adults | Gender: Female | ||||||||||||
Trial protocol: GB (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2008-007158-36 | Sponsor Protocol Number: GAU-CL-202X | Start Date*: 2009-02-25 | |||||||||||
Sponsor Name:Amicus Therapeutics, Inc. | |||||||||||||
Full Title: An open-label, multicentre, long-term extension study to assess the safety, efficacy and pharmacodynamics of AT2101 in adult patients with Type I Gaucher disease. | |||||||||||||
Medical condition: Type I Gaucher Disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2019-000667-24 | Sponsor Protocol Number: ST-920-201 | Start Date*: 2019-11-12 | |||||||||||
Sponsor Name:Sangamo Therapeutics, Inc. | |||||||||||||
Full Title: A Phase I/II, Multicenter, Open-Label, SingleDose, Dose-Ranging Study to Assess the Safety and Tolerability of ST-920, a rAAV2/6 Human Alpha Galactosidase A Gene Therapy in Subjects with Fabry Dise... | |||||||||||||
Medical condition: Fabry Disease (X-linked lysosomal storage disease) | |||||||||||||
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Population Age: Adults | Gender: Male | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) DE (Trial now transitioned) IT (Trial now transitioned) | |||||||||||||
Trial results: (No results available) |
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