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Clinical trials for CRISPR

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   44363   clinical trials with a EudraCT protocol, of which   7387   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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    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
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    5 result(s) found for: CRISPR. Displaying page 1 of 1.
    EudraCT Number: 2019-003462-40 Sponsor Protocol Number: 18IC07 Start Date*: 2020-03-30
    Sponsor Name:Great Ormond Street Hospital for Children NHS Trust
    Full Title: Phase 1 , open label study of CRISPR-CAR genome edited T cells (TT52CAR19) in relapsed /refractory B Cell Acute Lymphoblastic Leukaemia
    Medical condition: Relapsed/ refractory B- cell acute lymphoid leukaemia
    Disease:
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-001693-33 Sponsor Protocol Number: ITL-2002-CL-001 Start Date*: 2022-03-30
    Sponsor Name:Intellia Therapeutics, Inc.
    Full Title: Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of NTLA-2002 in Adults with Hereditary Angioedema (HAE)
    Medical condition: Hereditary Angioedema
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) FR (Trial now transitioned) DE (Trial now transitioned) ES (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2017-003351-38 Sponsor Protocol Number: CTX001-111 Start Date*: 2018-07-25
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion-Dependen...
    Medical condition: Transfusion-Dependent β Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) IT (Completed) GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2021-006390-37 Sponsor Protocol Number: VX21-CTX001-161 Start Date*: Information not available in EudraCT
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Transfusion-Depend...
    Medical condition: Transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD)
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040641 Sickle cell anaemia PT
    20.1 10010331 - Congenital, familial and genetic disorders 10054660 Thalassemia beta LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-001320-19 Sponsor Protocol Number: CTX001-121 Start Date*: 2018-12-05
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 1/2/3 Study to Evaluate the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects With Severe Sick...
    Medical condition: Severe Sickle Cell Disease (SCD)
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040641 Sickle cell anaemia PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) GB (GB - no longer in EU/EEA) DE (Completed) IT (Trial now transitioned)
    Trial results: (No results available)
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    Query did not match any studies.
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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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