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Clinical trials for X-linked severe combined immunodeficiency

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   44335   clinical trials with a EudraCT protocol, of which   7366   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 ).  
     
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    5 result(s) found for: X-linked severe combined immunodeficiency. Displaying page 1 of 1.
    EudraCT Number: 2018-000673-68 Sponsor Protocol Number: 16IC17 Start Date*: 2018-10-09
    Sponsor Name:Great Ormond Street Hospital for Children NHS Trust
    Full Title: Phase I/II study of lentiviral gene transfer for SCID-X1 with low dose targeted busulfan
    Medical condition: Severe combined immunodeficiency disorder (SCID) is a heterogeneous group of inherited disorders characterized by a profound reduction or absence of T lymphocyte function, resulting in lack of both...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10069566 Severe combined immunodeficiency syndrome LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: (No results available)
    EudraCT Number: 2021-003221-30 Sponsor Protocol Number: D5290C00008 Start Date*: 2021-10-22
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase 2, Open-label, Uncontrolled, Single-dose Study to Evaluate the Safety and Tolerability, Pharmacokinetics, and Occurrence of Antidrug Antibody for Nirsevimab in Immunocompromised Children ≤ ...
    Medical condition: RSV disease
    Disease: Version SOC Term Classification Code Term Level
    21.1 100000004865 10066742 Respiratory syncytial virus infection prophylaxis LLT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: ES (Ongoing) BE (Completed) PL (Completed)
    Trial results: View results
    EudraCT Number: 2007-000684-16 Sponsor Protocol Number: 03MI10 Start Date*: 2010-01-21
    Sponsor Name:Great Ormond Street Hospital NHS Trust / University College London - Institute of Child Health
    Full Title: Gene therapy for SCID-X1 using a self-inactivating (SIN) gammaretroviral vector.
    Medical condition: X-Linked severe combined Immunodeficiency (SCID-X1)
    Disease: Version SOC Term Classification Code Term Level
    9.1 10010099 Combined immunodeficiency LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2014-004491-31 Sponsor Protocol Number: M12-420 Start Date*: 2015-02-03
    Sponsor Name:Abbott Japan Co., Ltd
    Full Title: Multi-center, Open-label, Uncontrolled Clinical Study of Palivizumab in Japanese Newborns, Infants and Young Children at the Age of 24 Months or Less with Immunocompromised Medical Conditions
    Medical condition: Respiratory Syncytial Virus Infection
    Disease: Version SOC Term Classification Code Term Level
    17.1 10021881 - Infections and infestations 10061603 Respiratory syncytial virus infection PT
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-002004-10 Sponsor Protocol Number: 15-007 Start Date*: 2016-10-13
    Sponsor Name:Jazz Pharmaceuticals, Inc.
    Full Title: A Phase 3, Randomized, Adaptive Study Comparing the Efficacy and Safety of Defibrotide vs Best Supportive Care in the Prevention of Hepatic Veno-Occlusive Disease in Adult and Pediatric Patients Un...
    Medical condition: Prevention of Hepatic Veno-Occlusive Disease following Hematopoietic Stem Cell Transplant
    Disease: Version SOC Term Classification Code Term Level
    19.0 10019805 - Hepatobiliary disorders 10047207 Veno-occlusive liver damage LLT
    19.0 10019805 - Hepatobiliary disorders 10047217 Venoocclusive syndrome of the liver LLT
    19.0 10019805 - Hepatobiliary disorders 10047216 Venoocclusive liver disease PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) GB (Completed) BE (Completed) DE (Prematurely Ended) FR (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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