- Trials with a EudraCT protocol (18)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
18 result(s) found for: pompe disease late onset.
Displaying page 1 of 1.
EudraCT Number: 2019-003595-38 | Sponsor Protocol Number: AT845-01 | Start Date*: 2020-11-20 | |||||||||||
Sponsor Name:Audentes Therapeutics, Inc. | |||||||||||||
Full Title: FORTIS: A Phase 1/2, Open-Label, Ascending-Dose Clinical Study to Evaluate the Safety and Preliminary Efficacy of AT845, an AAV8-Delivered Gene Transfer Therapy in Patients with Late Onset Pompe Di... | |||||||||||||
Medical condition: Late Onset Pompe Disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2019-002251-42 | Sponsor Protocol Number: GZ-2016-11512. | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name: | |||||||||||||
Full Title: An open-label, single-center study on the safety and efficacy of avalglucosidase alpha in late-onset Pompe patients ≥5 years | |||||||||||||
Medical condition: Late onset Pompe disease | |||||||||||||
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Population Age: Adolescents, Under 18, Adults | Gender: Male, Female | ||||||||||||
Trial protocol: NL (Ongoing) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2019-001283-30 | Sponsor Protocol Number: SPK-3006-101 | Start Date*: 2020-06-16 | |||||||||||
Sponsor Name:Spark Therapeutics | |||||||||||||
Full Title: Phase 1/2, dose-escalation study to evaluate the safety, tolerability and efficacy of a single intravenous infusion of SPK-3006 in adults with late-onset Pompe disease | |||||||||||||
Medical condition: Pompe Disease (also known as glycogen storage disease type II) | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Prematurely Ended) DE (Ongoing) NL (Ongoing) DK (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2014-002158-38 | Sponsor Protocol Number: 701-201 | Start Date*: 2015-03-23 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc | |||||||||||||
Full Title: A Study of Respiratory Muscle Strength, including Effort-Independent Measures, in Subjects with Late-Onset Pompe Disease | |||||||||||||
Medical condition: Pompe disease | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Ongoing) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2013-001768-48 | Sponsor Protocol Number: 701-301 | Start Date*: 2013-11-27 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc | |||||||||||||
Full Title: A Phase 3 Switchover Study of the Efficacy and Safety of BMN 701 (GILT-tagged Recombinant Human GAA) and Long-Term Study for Extended Treatment in rhGAA Exposed Subjects with Late-onset Pompe Disease | |||||||||||||
Medical condition: Pompe disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Prematurely Ended) BE (Prematurely Ended) DE (Prematurely Ended) IT (Completed) PT (Prematurely Ended) AT (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2010-020611-36 | Sponsor Protocol Number: AGLU07310 | Start Date*: 2011-10-12 | |||||||||||
Sponsor Name:Genzyme Europe B.V. | |||||||||||||
Full Title: A Phase 4 Prospective Exploratory Muscle Biopsy, Biomarker, and Imaging Assessment Study in Patients With Late-Onset Pompe Disease Treated With Alglucosidase Alfa | |||||||||||||
Medical condition: Pompe disease (acid alpha-glucosidase deficiency) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) DE (Completed) NL (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2016-000942-77 | Sponsor Protocol Number: EFC14028 | Start Date*: 2016-07-15 | |||||||||||
Sponsor Name:Genzyme Corporation | |||||||||||||
Full Title: A Phase 3 Randomized, Multicenter, Multinational, Double-blinded Study Comparing the Efficacy and Safety of Repeated Biweekly Infusions of Avalglucosidase Alfa (neoGAA, GZ402666) and Alglucosidase ... | |||||||||||||
Medical condition: Pompe disease (acid alpha-glucosidase deficiency) | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (GB - no longer in EU/EEA) DK (Prematurely Ended) SE (Prematurely Ended) NL (Completed) ES (Ongoing) CZ (Completed) DE (Completed) BE (Completed) AT (Completed) PL (Completed) IT (Completed) PT (Completed) BG (Completed) HU (Completed) Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2006-003644-31 | Sponsor Protocol Number: AGLU03206 | Start Date*: 2007-01-18 |
Sponsor Name:Genzyme Europe BV | ||
Full Title: An Open-Label Extension Study of Patients with Late-Onset Pompe Disease Who Were Previously Enrolled in Protocol AGLU02704 | ||
Medical condition: Glycogen Storage Disease type II (Pompe's disease) | ||
Disease: | ||
Population Age: Adults, Elderly | Gender: Male, Female | |
Trial protocol: FR (Completed) NL (Completed) DE (Completed) | ||
Trial results: View results |
EudraCT Number: 2021-002590-26 | Sponsor Protocol Number: PTA17333 | Start Date*: Information not available in EudraCT | |||||||||||
Sponsor Name:Sanofi-Aventis Recherche & Développement | |||||||||||||
Full Title: A French multicenter Phase 4 open label extension study of long term safety and efficacy in patients with Pompe disease who previously participated in avalglucosidase development studies in France | |||||||||||||
Medical condition: Glycogen storage disease type II | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Ongoing) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2010-023561-22 | Sponsor Protocol Number: POM-001 | Start Date*: 2011-08-15 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc | |||||||||||||
Full Title: A Phase 1/2 Open-label Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamic and Preliminary Efficacy of BMN 701 (GILT-tagged Recombinant human GAA) in Patients with Late-onset Pompe... | |||||||||||||
Medical condition: Pompe disease | |||||||||||||
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Population Age: Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) DE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2005-002759-42 | Sponsor Protocol Number: AGLU02704 | Start Date*: 2007-07-18 | |||||||||||
Sponsor Name:Genzyme Europe BV | |||||||||||||
Full Title: A Randomized, Double-Blind, Multicenter, Multinational, Placebo-Controlled Study of the Safety, Efficacy, and Pharmacokinetics of Myozyme, Recombinant Human Acid alpha-Glucosidase (rhGAA), Treatmen... | |||||||||||||
Medical condition: Glycogen Storage Disease type II (Pompe's disease) | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: DE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2017-004370-34 | Sponsor Protocol Number: POM-003 | Start Date*: 2018-04-09 | |||||||||||
Sponsor Name:Amicus Therapeutics, Inc. | |||||||||||||
Full Title: A Prospective Study in Subjects with Late-onset Pompe Disease who are Currently Being Treated with Enzyme Replacement Therapy | |||||||||||||
Medical condition: Late-onset Pompe disease (LOPD) in subjects receiving standard-of-care enzyme replacement therapy (ERT) | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: BE (Prematurely Ended) HU (Prematurely Ended) | |||||||||||||
Trial results: View results |
EudraCT Number: 2011-001805-28 | Sponsor Protocol Number: POM-002 | Start Date*: 2012-07-03 | |||||||||||
Sponsor Name:BioMarin Pharmaceutical Inc | |||||||||||||
Full Title: A Long-Term Study for Extended BMN 701Treatment of Patients with Pompe Disease who have Participated in a BMN 701 Study | |||||||||||||
Medical condition: Pompe disease | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) DE (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2005-002829-31 | Sponsor Protocol Number: AGLU02804, Amendment 1 | Start Date*: 2005-08-31 |
Sponsor Name:Genzyme Europe BV | ||
Full Title: Full title of the trial : A Single Centre, Open-Label, Bridging Study of the Safety, Pharmacokinetics and Efficacy of Recombinant Human Acid Alpha-Glucosidase (rhGAA) Treatment in Patients with Lat... | ||
Medical condition: Glycogen Storage Disease type II (Pompe´s disease) | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | |
Trial protocol: GB (Completed) BE (Completed) | ||
Trial results: View results |
EudraCT Number: 2016-004578-16 | Sponsor Protocol Number: VAL-1221-201-16 | Start Date*: 2017-06-19 | ||||||||||||||||
Sponsor Name:Valerion Therapeutics, LLC | ||||||||||||||||||
Full Title: A Three-month, Open-Label, Randomized, Dose-escalation Study of the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics and Preliminary Efficacy of VAL-1221 versus Myozyme®/Lumizyme® in Patien... | ||||||||||||||||||
Medical condition: Late-Onset GSD-II (Pompe Disease) | ||||||||||||||||||
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Population Age: Adults | Gender: Male, Female | |||||||||||||||||
Trial protocol: GB (Prematurely Ended) | ||||||||||||||||||
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 (Ongoing) FR (Ongoing) BE (Ongoing) GR (Ongoing) SI (Ongoing) AT (Completed) IT (Ongoing) | |||||||||||||
Trial results: (No results available) |
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: 2008-005979-97 | Sponsor Protocol Number: 0816202 | Start Date*: 2009-01-27 | |||||||||||
Sponsor Name:CHU de Toulouse | |||||||||||||
Full Title: EFFICACITE DU TRAITEMENT PAR POMPE A INSULINE EXTERNE CHEZ LE DIABETIQUE DE TYPE 2 : ETUDE RANDOMISEE EN PARALLELE SUR 1 AN VERSUS MULTI-INJECTIONS | |||||||||||||
Medical condition: diabète de type II | |||||||||||||
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Population Age: Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: FR (Ongoing) | |||||||||||||
Trial results: (No results available) |
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