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Clinical trials for Regulatory DNA sequences

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
  • interventional clinical trials that were approved in the European Union (EU)/European Economic Area (EEA) under the Clinical Trials Directive 2001/20/EC
  • clinical trials conducted outside the EU/EEA that are linked to European paediatric-medicine development

    • EU/EEA interventional clinical trials approved under or transitioned to the Clinical Trial Regulation 536/2014 are publicly accessible through the
    Clinical Trials Information System (CTIS).

    The EU Clinical Trials Register currently displays   44365   clinical trials with a EudraCT protocol, of which   7389   are clinical trials conducted with subjects less than 18 years old.   The register also displays information on   18700   older paediatric trials (in scope of Article 45 of the Paediatric Regulation (EC) No 1901/2006).

    Phase 1 trials conducted solely on adults and that are not part of an agreed paediatric investigation plan (PIP) are not publicly available (see Frequently Asked Questions ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
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    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
    Disease: Version SOC Term Classification Code Term Level
    18.1 10010331 - Congenital, familial and genetic disorders 10075697 Gaucher's disease type I PT
    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
    Disease: Version SOC Term Classification Code Term Level
    27.1 10010331 - Congenital, familial and genetic disorders 10070668 Huntington's disease PT
    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
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    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: View results
    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
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10057080 Homozygous familial hypercholesterolemia LLT
    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
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    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
    Disease: Version SOC Term Classification Code Term Level
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10042863 Synovial sarcoma PT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10061873 Non-small cell lung cancer PT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10028228 Multiple myeloma LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10073137 Myxoid liposarcoma PT
    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
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018048 Gaucher's disease LLT
    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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    The status and protocol content of GB trials is no longer updated since 1 January 2021. For the UK, as of 31 January 2021, EU Law applies only to the territory of Northern Ireland (NI) to the extent foreseen in the Protocol on Ireland/NI. Legal notice
    As of 31 January 2023, all EU/EEA initial clinical trial applications must be submitted through CTIS . Updated EudraCT trials information and information on PIP/Art 46 trials conducted exclusively in third countries continues to be submitted through EudraCT and published on this website.

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