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Clinical trials for Combined immunodeficiencies

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   44339   clinical trials with a EudraCT protocol, of which   7369   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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    7 result(s) found for: Combined immunodeficiencies. 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: 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: 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: 2018-001029-14 Sponsor Protocol Number: P150949J Start Date*: 2018-10-25
    Sponsor Name:ASSISTANCE PUBLIQUE - HOPITAUX DE PARIS (AP-HP)
    Full Title: A phase I/II Study evaluating the safety and the efficacy of Human T Lymphoid Progenitor (HTLP) injection to accelerate immune reconstitution after partially HLA compatible allogeneic hematopoietic...
    Medical condition: SCID pediatric patients (n=12 for analysis) requiring an HLA partially compatible allogeneic HSCT.
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010099 Combined immunodeficiency PT
    Population Age: Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: FR (Prematurely Ended) DE (Ongoing) IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2013-005508-33 Sponsor Protocol Number: MC-FludT.16/NM Start Date*: 2014-09-29
    Sponsor Name:medac Gesellschaft für klinische Spezialpräparate mbH
    Full Title: Clinical phase II trial to compare Treosulfan-based conditioning therapy with Busulfan-based conditioning prior to allogeneic haematopoietic stem cell transplantation (HSCT) in paediatric patients ...
    Medical condition: Male and female children with non-malignant diseases requiring myeloablative conditioning treatment with following allogeneic haematopoietic stem cell transplantation (allo-HSCT) – i.e. primary imm...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10021606 Inborn errors of metabolism NEC HLT
    20.0 100000004870 10036700 Primary immunodeficiency syndromes HLT
    20.0 100000004850 10018903 Haemoglobinopathies congenital HLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Completed) AT (Completed) CZ (Completed) PL (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2019-002343-14 Sponsor Protocol Number: RAG1-2019-01 Start Date*: 2020-09-25
    Sponsor Name:Leiden University Medical Center
    Full Title: PHASE I/II CLINICAL TRIAL OF AUTOLOGOUS HEMATOPOIETIC STEM CELL GENE THERAPY FOR RAG1-DEFICIENT SEVERE COMBINED IMMUNODEFICIENCY
    Medical condition: Patients with severe combined immunodeficiency (SCID) based on a genetic defect in the Recombinase Activating Gene 1 (RAG1)
    Disease:
    Population Age: Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) ES (Ongoing) PL (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2011-005562-38 Sponsor Protocol Number: M2011-238 Start Date*: 2012-11-13
    Sponsor Name:Miltenyi Biotec GmbH
    Full Title: A multi-center phase I/II safety and feasibility study using CliniMACS TCRα/β and CD19 depleted stem cell grafts from haploidentical donors for haematopoietic progenitor cell transplantation in chi...
    Medical condition: Hematological and non-hematological malignancies, and non-malignant diseases, requiring allogeneic blood stem cell transplantation without available HLA-identical donor.
    Disease: Version SOC Term Classification Code Term Level
    18.1 10042613 - Surgical and medical procedures 10063581 Stem cell transplant PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) NL (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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