- Trials with a EudraCT protocol (8)
- Paediatric studies in scope of Art45 of the Paediatric Regulation (0)
8 result(s) found for: Regulatory DNA sequences.
Displaying page 1 of 1.
EudraCT Number: 2012-003427-38 | Sponsor Protocol Number: HGT-GCB-068 | Start Date*: 2016-04-07 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies | |||||||||||||
Full Title: A Multi-center, Open-label, Efficacy and Safety Study of Velaglucerase Alfa Enzyme Replacement Therapy in Children and Adolescents With Type 3 Gaucher Disease | |||||||||||||
Medical condition: Type 3 Gaucher disease | |||||||||||||
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Population Age: Children, Adolescents, Under 18 | Gender: Male, Female | ||||||||||||
Trial protocol: Outside EU/EEA | |||||||||||||
Trial results: View results |
EudraCT Number: 2020-001461-36 | Sponsor Protocol Number: CT-AMT-130-02 | Start Date*: 2021-06-30 | |||||||||||
Sponsor Name:uniQure biopharma B.V. | |||||||||||||
Full Title: A Phase Ib/II Randomised, Double-Blind Study to Explore Safety, Tolerability, and Efficacy Signals of Multiple Doses of Striatally-Administered rAAV5-miHTT Total Huntingtin Gene (HTT) Lowering Ther... | |||||||||||||
Medical condition: Huntington Disease | |||||||||||||
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Population Age: Adults | Gender: Male, Female | ||||||||||||
Trial protocol: PL (Trial now transitioned) DE (Prematurely Ended) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2008-001965-27 | Sponsor Protocol Number: HGT-GCB-044 | Start Date*: 2008-10-29 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies, Inc. | |||||||||||||
Full Title: An Open-Label Extension Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients with Type 1 Gaucher Disease | |||||||||||||
Medical condition: Type I Gaucher disease | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) ES (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2022-002323-35 | Sponsor Protocol Number: TAK-669-3001 | Start Date*: 2022-11-10 |
Sponsor Name: Takeda Development Center Americas, Inc. | ||
Full Title: A Multicenter, Open-label Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of Velaglucerase Alfa in Chinese Subjects With Type 1 Gaucher Disease | ||
Medical condition: Type 1 Gaucher Disease | ||
Disease: | ||
Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | |
Trial protocol: Outside EU/EEA | ||
Trial results: (No results available) |
EudraCT Number: 2019-003611-62 | Sponsor Protocol Number: LIB003-003 | Start Date*: 2020-03-20 | |||||||||||
Sponsor Name:LIB Therapeutics, LLC | |||||||||||||
Full Title: Randomized, Open-Label, Cross-Over, Phase 3 Study to Evaluate the Efficacy and Safety of LIB003 with Evolocumab in Homozygous Familial Hypercholesterolemia Patients on Stable Lipid-Lowering Therapy. | |||||||||||||
Medical condition: Homozygous Familial Hypercholesterolemia | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: NO (Completed) | |||||||||||||
Trial results: (No results available) |
EudraCT Number: 2006-006304-11 | Sponsor Protocol Number: TKT034 | Start Date*: 2007-07-03 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies Inc | |||||||||||||
Full Title: A multicenter open-label study of Gene-Activated Human Glucocerebrosidase (GA-GCB) enzyme replacement therapy in patients with type 1 Gaucher disease previously treated wiht imiglucerase | |||||||||||||
Medical condition: Type I Gaucher Disease | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: GB (Completed) DE (Completed) IT (Completed) ES (Completed) | |||||||||||||
Trial results: View results |
EudraCT Number: 2018-004888-31 | Sponsor Protocol Number: 208750 | Start Date*: 2020-01-27 | ||||||||||||||||||||||||||
Sponsor Name:GlaxoSmithKline research & development Ltd. | ||||||||||||||||||||||||||||
Full Title: Long-Term Follow-Up (LTFU) of Participants Treated with GSK Adoptive Cell Therapies | ||||||||||||||||||||||||||||
Medical condition: Synovial sarcoma Multiple myeloma Non-small cell lung cancer Myxoid liposarcoma | ||||||||||||||||||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | |||||||||||||||||||||||||||
Trial protocol: NL (Trial now transitioned) SE (Completed) IT (Trial now transitioned) FR (Prematurely Ended) | ||||||||||||||||||||||||||||
Trial results: (No results available) |
EudraCT Number: 2007-002840-21 | Sponsor Protocol Number: HGT-GCB-039 | Start Date*: 2007-10-30 | |||||||||||
Sponsor Name:Shire Human Genetic Therapies Inc | |||||||||||||
Full Title: A Multicenter, Randomized, Double-Blind, Parallel-Group Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy Compared with Imiglucerase in Patients with Type I Gauc... | |||||||||||||
Medical condition: Type I Gaucher disease Enfermedad de Gaucher tipo 1 | |||||||||||||
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Population Age: Children, Adolescents, Under 18, Adults, Elderly | Gender: Male, Female | ||||||||||||
Trial protocol: ES (Completed) GB (Completed) IT (Completed) | |||||||||||||
Trial results: View results |
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