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Clinical trials for Wiskott-Aldrich Syndrome

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   44334   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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    11 result(s) found for: Wiskott-Aldrich Syndrome. Displaying page 1 of 1.
    EudraCT Number: 2007-004308-11 Sponsor Protocol Number: GTG002.07 Start Date*: 2010-01-11
    Sponsor Name:Genethon
    Full Title: PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME
    Medical condition: Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopenia,...
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10047992 Wiskott-Aldrich syndrome PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2014-000274-20 Sponsor Protocol Number: GNT-WAS-03 Start Date*: 2014-05-14
    Sponsor Name:Genethon
    Full Title: LONG TERM SAFETY FOLLOW UP OF PATIENTS ENROLLED IN THE PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME(GTG 002-07 AND GTG 003-08)
    Medical condition: Wiskott-Aldrich syndrome (WAS) is a rare X-linked immunodeficiency caused by mutations in a single gene ,the Wiskott-Aldrich Syndrome Protein (WASP). WAS is characterised by micro-thrombocytopeni...
    Disease: Version SOC Term Classification Code Term Level
    19.1 10010331 - Congenital, familial and genetic disorders 10047992 Wiskott-Aldrich syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: GB (GB - no longer in EU/EEA) FR (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2009-011152-22 Sponsor Protocol Number: GTG003.08 Start Date*: Information not available in EudraCT
    Sponsor Name:GENETHON
    Full Title: Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome
    Medical condition: Phase 1/2 clinical trial of haematopoietic stem cell gene therapy for the Wiskott-Aldrich Syndrome. An open labelled, non-randomised, phase I/II, cohort study involving a single infusion of autolo...
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2004-004465-15 Sponsor Protocol Number: Study No. 957 Start Date*: 2005-04-05
    Sponsor Name:Biotest AG
    Full Title: A multicentre, open, prospective study investigating clinical efficacy, safety, and pharmacokinetic properties of the human normal immunoglobulin for intravenous administration BT681 in patients wi...
    Medical condition: primary immunodeficiency syndrome as congenital agammaglobulinaemia or hypogammaglobulinaemia, common variable immunodeficiency, severe combined immunodeficiencies, Wiskott Aldrich syndrome
    Disease: Version SOC Term Classification Code Term Level
    7.0 10036700 HLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) HU (Completed)
    Trial results: View results
    EudraCT Number: 2018-003842-18 Sponsor Protocol Number: OTL-103-4 Start Date*: 2019-01-08
    Sponsor Name:Orchard Therapeutics Ltd.
    Full Title: A Single Arm, Open Label Clinical Study of Haematopoietic Stem Cell Gene Therapy with Cryopreserved Autologous CD34+ Cells Transduced with Lentiviral Vector encoding WAS cDNA in Subjects with Wisko...
    Medical condition: Wiskott-Aldrich Syndrome
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021428 - Immune system disorders 10061598 Immunodeficiency PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: IT (Trial now transitioned)
    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: 2009-017346-32 Sponsor Protocol Number: 201228 Start Date*: 2010-03-15
    Sponsor Name:Fondazione Telethon
    Full Title: A PHASE I/II CLINICAL TRIAL OF HAEMATOPOIETIC STEM CELL GENE THERAPY FOR THE WISKOTT-ALDRICH SYNDROME
    Medical condition: Wiskott-Aldrich Syndrom
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021428 - Immune system disorders 10061598 Immunodeficiency PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    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: 2011-005679-18 Sponsor Protocol Number: GMX04 Start Date*: 2011-12-07
    Sponsor Name:Bio Products Laboratory Limited
    Full Title: A Phase IV, Multicenter, Open-Label Study to Evaluate the Efficacy, Safety and Pharmacokinetics of Gammaplex in Primary Immunodeficiency Diseases (PID) in Children and Adolescents
    Medical condition: Primary immunodeficiency diseases
    Disease: Version SOC Term Classification Code Term Level
    14.0 10005329 - Blood and lymphatic system disorders 10005329 Blood and lymphatic system disorders SOC
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: Removed from public view
    EudraCT Number: 2014-000608-90 Sponsor Protocol Number: Eltrombo-IT-2014 Start Date*: 2014-12-15
    Sponsor Name:Fondazione IRCCS Policlinico San Matteo
    Full Title: Eltrombopag for inherited thrombocytopenias.
    Medical condition: Inherited thrombocytopenias
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004851 10043555 Thrombocytopenias HLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2022-002378-95 Sponsor Protocol Number: D7000C00001 Start Date*: 2023-06-21
    Sponsor Name:AstraZeneca AB
    Full Title: A Phase I/III Randomized, Double-blind Study to Evaluate the Safety, Efficacy, and Neutralizing Activity of AZD5156/AZD3152 for Pre-exposure Prophylaxis of COVID-19 in Participants with Conditions ...
    Medical condition: COVID-19
    Disease: Version SOC Term Classification Code Term Level
    23.0 10021881 - Infections and infestations 10051905 Coronavirus infection PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Trial now transitioned) DK (Trial now transitioned) DE (Trial now transitioned) PL (Trial now transitioned) ES (Ongoing) FR (Trial now transitioned) SE (Prematurely Ended)
    Trial results: (No results available)
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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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