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Clinical trials for Severe Combined Immunodeficiency

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    The EU Clinical Trials Register currently displays   43851   clinical trials with a EudraCT protocol, of which   7283   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).

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    21 result(s) found for: Severe Combined Immunodeficiency. Displaying page 1 of 2.
    1  2  Next»
    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: 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: 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: 2019-003555-11 Sponsor Protocol Number: PD20180302 Start Date*: Information not available in EudraCT
    Sponsor Name:ASSISTANCE PUBLIQUE-HOPÏTAUX DE PARIS
    Full Title: A Phase 1/2 Open Label non randomized Study, multicentric, single arm evaluating the Safety and Efficacy of Gene Therapy of the severe combined immunodeficiency (SCID) caused by mutations in the ...
    Medical condition: immunodeficiency (SCID) caused by mutations in the human DCLRE1C gene (Artemis)
    Disease:
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: FR (Ongoing)
    Trial results: (No results available)
    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 (Ongoing) ES (Ongoing) PL (Ongoing)
    Trial results: (No results available)
    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 (Ongoing) DE (Ongoing) IT (Ongoing)
    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: 2017-001275-23 Sponsor Protocol Number: OTL-101-5(17IC04) Start Date*: 2017-09-21
    Sponsor Name:Great Ormond Street Hospital for Children NHS Trust
    Full Title: Efficacy and safety of a cryopreserved formulation of autologous CD34+ haematopoietic stem cells transduced ex vivo with EFS lentiviral vector encoding for human ADA gene in subjects with Severe Co...
    Medical condition: Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protect...
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10066372 ADA deficiency LLT
    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: 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: 2017-001731-39 Sponsor Protocol Number: STRIM002-(205813) Start Date*: 2017-10-12
    Sponsor Name:Orchard Therapeutics (Europe) Ltd
    Full Title: Methodology study to investigate the utility of retroviral insertion site analysis in samples from subjects treated with Strimvelis™ gene therapy
    Medical condition: Adenosine deaminase (ADA) deficiency severe combined immunodeficiency
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10066367 Adenosine deaminase deficiency LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2022-003268-25 Sponsor Protocol Number: AT-03A-017 Start Date*: 2023-05-12
    Sponsor Name:Atea Pharmaceuticals, Inc.
    Full Title: A Phase 3 Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Bemnifosbuvir in High-Risk Outpatients with COVID-19
    Medical condition: COVID-19
    Disease: Version SOC Term Classification Code Term Level
    23.0 10021881 - Infections and infestations 10084268 COVID-19 PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2010-024253-36 Sponsor Protocol Number: 10MI29 Start Date*: 2012-08-03
    Sponsor Name:Great Ormond Street Hospital for Children NHS Trust
    Full Title: Phase I/II, historical controlled, open-label, non-randomised, single-centre trial to assess the safety and efficacy of EF1αS-ADA lentiviral vector mediated gene modification of autologus CD34+ cel...
    Medical condition: Adenosine deaminase (ADA) deficiency is an inherited disorder that damages the immune system and causes severe combined immunodeficiency (SCID). Children with SCID lack virtually all immune protect...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000012248 10066372 ADA deficiency LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2019-000660-25 Sponsor Protocol Number: CT-P17_3.2 Start Date*: 2019-07-09
    Sponsor Name:CELLTRION, Inc.
    Full Title: A Phase III, Open-label, Single-arm, Multiple-dose Study to Evaluate Usability of Subcutaneous Auto-injector of CT-P17 in Patients with Moderate to Severe Active Rheumatoid Arthritis
    Medical condition: Moderate to Severe Active Rheumatoid Arthritis
    Disease: Version SOC Term Classification Code Term Level
    21.0 10028395 - Musculoskeletal and connective tissue disorders 10039073 Rheumatoid arthritis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: PL (Completed)
    Trial results: View results
    EudraCT Number: 2019-002267-10 Sponsor Protocol Number: MK-5592-104 Start Date*: 2019-12-17
    Sponsor Name:Merck Sharp & Dohme Corp., a subsidiary of Merck & Co., Inc.
    Full Title: A Phase 2, Open-Label, Non-Comparative Clinical Trial to Study the Safety and Efficacy of Posaconazole (POS, MK-5592) in Pediatric Participants Aged 2 to <18 Years With Invasive Aspergillosis
    Medical condition: Invasive aspergillosis (IA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004862 10003488 Aspergillosis LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: HU (Completed) Outside EU/EEA GR (Completed) BE (Completed) IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-002643-18 Sponsor Protocol Number: REDUCE Start Date*: 2018-09-04
    Sponsor Name:Charité Universitätsmedizin Berlin
    Full Title: REDUCE - Multicenter, prospective, randomized study investigating the efficacy and safety of a reduced immunosuppressive therapy with tacrolimus once daily in comparison to standard triple immunosu...
    Medical condition: Immunosuppression after kidney transplantion in elderly patients ≥65 years of age
    Disease: Version SOC Term Classification Code Term Level
    20.1 10021428 - Immune system disorders 10062016 Immunosuppression PT
    20.0 10021428 - Immune system disorders 10054990 Immunodeficiency secondary to organ transplantation LLT
    20.0 100000004863 10074474 Transplantation complications HLT
    Population Age: Elderly Gender: Male, Female
    Trial protocol: FR (Ongoing) GB (GB - no longer in EU/EEA) AT (Prematurely Ended) ES (Prematurely Ended) DK (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2021-000382-32 Sponsor Protocol Number: REP0121 Start Date*: 2021-08-16
    Sponsor Name:Dompé farmaceutici s.p.a.
    Full Title: A multicenter, randomized, double-blind, placebo-controlled phase 2 study to assess the efficacy and safety of oral reparixin in cancer related fatigue in patients with locally advanced or metastat...
    Medical condition: Cancer related fatigue in patients with locally advanced or metastatic breast cancer
    Disease: Version SOC Term Classification Code Term Level
    22.0 100000004864 10082262 Chemotherapy fatigue LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2021-006621-22 Sponsor Protocol Number: COVIC-19-G Start Date*: 2022-02-14
    Sponsor Name:DRK-Blutspendedienst Baden-Württemberg - Hessen gGmbH
    Full Title: A Randomised Open-Label Trial of Early, Very High-Titre Convalescent Plasma Therapy in Clinically Vulnerable Individuals with Mild COVID-19 as model of early treatment in a pandemia with a new path...
    Medical condition: Clinically Vulnerable Individuals with Mild COVID-19
    Disease: Version SOC Term Classification Code Term Level
    23.1 10022891 - Investigations 10084355 COVID-19 virus test positive LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2018-002565-18 Sponsor Protocol Number: SCY-078-206 Start Date*: 2019-10-04
    Sponsor Name:SCYNEXIS, Inc.
    Full Title: A Multicenter, Randomized, Double-Blind Study to Evaluate the Safety and Efficacy of the Coadministration of SCY-078 with Voriconazole in Patients with Invasive Pulmonary Aspergillosis (SCYNERGIA)
    Medical condition: Patients with Invasive Pulmonary Aspergillosis
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004862 10059259 Pulmonary aspergillosis LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) BE (Ongoing) ES (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2018-001489-41 Sponsor Protocol Number: P00003466 Start Date*: 2018-07-02
    Sponsor Name:Karolinska Universitetssjukhuset
    Full Title: Radiation- and alkylator-free hematopoietic cell transplantation for bone marrow failure due to dyskeratosis congenita / telomere disease
    Medical condition: Dyskeratosis congenita / telomere disease
    Disease: Version SOC Term Classification Code Term Level
    20.0 10042613 - Surgical and medical procedures 10001756 Allogenic bone marrow transplantation therapy PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: SE (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2019-003842-34 Sponsor Protocol Number: MK-5592-127 Start Date*: Information not available in EudraCT
    Sponsor Name:Merck Sharp & Dohme LLC
    Full Title: A Phase 2, Open-Label, Single-Arm, Sequential-Panel Study to Evaluate the Pharmacokinetics, Safety, and Tolerability of Posaconazole (POS, MK-5592) Intravenous and Powder for Oral Suspension Formul...
    Medical condition: Invasive Fungal Infection
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021881 - Infections and infestations 10017533 Fungal infection PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Under 18 Gender: Male, Female
    Trial protocol: PL (Ongoing) Outside EU/EEA BE (Trial now transitioned) GR (Ongoing)
    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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