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Clinical trials for Immune system disorders congenital

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   44412   clinical trials with a EudraCT protocol, of which   7421   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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    84 result(s) found for: Immune system disorders congenital. Displaying page 1 of 5.
    1  2  3  4  5  Next»
    EudraCT Number: 2020-003596-17 Sponsor Protocol Number: CMAS825D12201 Start Date*: 2021-03-09
    Sponsor Name:Novartis Pharma AG
    Full Title: A three-period multicenter study, with a randomized withdrawal, double-blinded, placebo-controlled design in Period 2 to evaluate the clinical efficacy, safety and tolerability of MAS825 in NLRC4-G...
    Medical condition: NLRC4-GOF, AIFEC (autoinflammation with infantile enterocolitis)
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10084306 Autoinflammation with infantile enterocolitis PT
    Population Age: Preterm newborn infants, Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) CZ (Trial now transitioned) IT (Trial now transitioned) ES (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2015-003418-25 Sponsor Protocol Number: CTU/2014/120 Start Date*: 2016-03-29
    Sponsor Name:MeiraGTx UK II Ltd
    Full Title: An Open-label, Multi-centre, Phase I/II Dose Escalation Trial of an Adeno-Associated Virus Vector (AAV2/5-OPTIRPE65) for Gene Therapy of Adults and Children with Retinal Dystrophy associated with D...
    Medical condition: Leber Congenital Amaurosis (LCA) caused by mutations in RPE65
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10070667 Leber's congenital amaurosis PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: View results
    EudraCT Number: 2007-004692-19 Sponsor Protocol Number: 963 Start Date*: 2007-12-19
    Sponsor Name:Biotest AG
    Full Title: Prevention of congenital cytomegalovirus infection in infants of mothers with primary cytomegalovirus infection during pregnancy. A randomised, open, controlled, multicentre and multinational study...
    Medical condition: Congenital CMV infection after primary CMV infection during pregnancy
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10010420 Congenital CMV infection LLT
    Population Age: In utero, Newborns, Infants and toddlers, Children, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) BE (Completed) HU (Completed) AT (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2015-005524-26 Sponsor Protocol Number: GBI1406 Start Date*: 2016-07-29
    Sponsor Name:Grifols Biologicals Inc.
    Full Title: A Multicenter Phase 2 Open-Label, Single-Arm, Prospective, Interventional Study of Plasma-Derived Factor VIIIIVWF Alphanate® in Immune Tolerance Induction Therapy in Subjects with Congenital Hemoph...
    Medical condition: Inhibitors in patients with severe Congenital Haemophilia A
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004851 10018847 Haematological disorders HLT
    Population Age: Newborns, Infants and toddlers, Children, Under 18 Gender: Male
    Trial protocol: ES (Prematurely Ended) IT (Completed)
    Trial results: View results
    EudraCT Number: 2005-004008-36 Sponsor Protocol Number: ANAK-01 Start Date*: 2005-10-27
    Sponsor Name:ISTITUTO GIANNINA GASLINI
    Full Title: Efficacy and safety of treatment with Anakinra in patients with CINCA/MUCKLE-WELLS syndrome and systemic juvenile idiopathic arthritis
    Medical condition: CINCA/MUCKLE-WELLS syndrome and systemic juvenile idiopathic arthritis
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2011-001118-32 Sponsor Protocol Number: EP06-401 Start Date*: 2011-05-25
    Sponsor Name:Sandoz GmbH
    Full Title: Twelve-month study on the immunogenicity, safety, and efficacy of Zarzio®/Filgrastim HEXAL® in patients with severe chronic neutropenia
    Medical condition: Severe chronic neutropenia (i.e. congenital neutropenia, cyclic neutropenia and chronic idiopathic neutropenia)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10005329 - Blood and lymphatic system disorders 10051645 Idiopathic neutropenia PT
    14.1 10010331 - Congenital, familial and genetic disorders 10069819 Congenital neutropenia LLT
    14.1 10005329 - Blood and lymphatic system disorders 10053176 Cyclic neutropenia PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Prematurely Ended) SE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-001678-40 Sponsor Protocol Number: CACZ885N2301E2 Start Date*: 2017-05-15
    Sponsor Name:Novartis pharma AG
    Full Title: An extension study of safety of canakinumab in Japanese patients with periodic fever syndromes
    Medical condition: Three disease of periodic fever syndromes, TNF receptor Associated Periodic Syndrome (TRAPS), Hyper IgDSyndrome (HIDS)/ Mevalonate Kinase Deficiency (MKD) and Familial Mediterranean Fever (FMF
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10010331 Congenital, familial and genetic disorders SOC
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2022-004155-13 Sponsor Protocol Number: BMS_IM101-931 Start Date*: 2023-04-20
    Sponsor Name:Universitätsklinikum Freiburg
    Full Title: Study to investigate the restoring of an immunological balance during therapy with abatacept Abatacept restores immune system equilibrium (ARISE)
    Medical condition: CVID patients confirmed according to ESID/PAGID criteria or related disorders which fulfill the diagnostic criteria for CVID and interstitial lung disease or granuloma diagnosed by chest CT positiv...
    Disease: Version SOC Term Classification Code Term Level
    20.1 10021428 - Immune system disorders 10021449 Immunodeficiency common variable PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: (No results available)
    EudraCT Number: 2014-003527-22 Sponsor Protocol Number: CINC424A2X01B Start Date*: 2015-04-30
    Sponsor Name:Novartis Farmacéutica, S.A.
    Full Title: An open label, multi-center, Phase IV roll-over protocol for patients who have completed a prior global Novartis or Incyte sponsored ruxolitinib (INC424) study and are judged by the investigator to...
    Medical condition: This roll-over study is designed to accept patients with varied disease origins. Please refer to the parent protocol for the disease background information and rationale for use of ruxolitinib in t...
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Completed) BE (Completed) DE (Trial now transitioned) GR (Completed) HU (Completed) SE (Trial now transitioned) PT (Completed) BG (Completed) FR (Completed) SK (Completed) DK (Completed) SI (Completed) IT (Trial now transitioned) PL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2016-003226-16 Sponsor Protocol Number: BP-404 Start Date*: 2017-02-03
    Sponsor Name:Bellicum Pharmaceuticals, Inc.
    Full Title: Follow-up of phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
    Medical condition: Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopath...
    Disease: Version SOC Term Classification Code Term Level
    19.0 10005329 - Blood and lymphatic system disorders 10018849 Haematological disorders NEC HLGT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2014-003605-15 Sponsor Protocol Number: IgPro20_3001 Start Date*: 2015-02-26
    Sponsor Name:CSL Behring AG
    Full Title: A Multicenter Extension Study of the Efficacy, Tolerability, and Safety of Immune Globulin Subcutaneous (Human) IgPro20 in Subjects With Primary Immunodeficiency (PID)
    Medical condition: Primary Immune Deficiency (PID)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10064859 Primary immunodeficiency syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2014-003607-30 Sponsor Protocol Number: ZLB04_009CR Start Date*: 2015-02-26
    Sponsor Name:CSL Behring AG
    Full Title: A Phase III Open-Label, Prospective, Multicenter Study of the Efficacy, Tolerability, Safety, and Pharmacokinetics of Immune Globulin Subcutaneous (Human), IgPro20 in Subjects With Primary Immunode...
    Medical condition: Primary Immune Deficiency
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10064859 Primary immunodeficiency syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2010-019249-25 Sponsor Protocol Number: 981 Start Date*: 2010-08-12
    Sponsor Name:Biotest AG
    Full Title: An open, prospective trial investigating pharmacokinetics and safety (Part A) of the human normal immunoglobulin for intravenous infusion (IVIG) BT090 and tolerability and safety of escalating infu...
    Medical condition: Primary immunodeficiency disease (PID)
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10010509 Congenital hypogammaglobulinemia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Completed) HU (Completed)
    Trial results: View results
    EudraCT Number: 2014-000584-41 Sponsor Protocol Number: BP-004 Start Date*: 2014-10-23
    Sponsor Name:Bellicum Pharmaceuticals, Inc.
    Full Title: Phase I/II study of CaspaCide T cells from an HLA-partially matched family donor after negative selection of TCR αβ+ T cells in pediatric patients affected by hematological disorders
    Medical condition: Hematological disorders (ALL;AML;Non-Hodgkin lymphoma;Myelodysplastic syndromes;Congenital immune deficiencies;Severe aplastic anemia;Fanconi anemia; Osteopetrosis;Selected cases of hemoglobinopath...
    Disease: Version SOC Term Classification Code Term Level
    16.1 10005329 - Blood and lymphatic system disorders 10018849 Haematological disorders NEC HLGT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed) GB (GB - no longer in EU/EEA) ES (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-001693-28 Sponsor Protocol Number: CONE-01 Start Date*: 2020-02-06
    Sponsor Name:Octapharma Pharmazeutika Produktionsges.m.b.H.
    Full Title: Prospective, open-label, single arm, multicenter, pharmacokinetic, and safety study of a single dose intravenous human plasma-derived C1 Esterase Inhibitor (C1-INH) concentrate in patients with con...
    Medical condition: Hereditary angioedema type I and type II
    Disease: Version SOC Term Classification Code Term Level
    23.1 10010331 - Congenital, familial and genetic disorders 10019860 Hereditary angioedema PT
    21.0 10010331 - Congenital, familial and genetic disorders 10080956 Hereditary angioedema type I LLT
    21.0 10010331 - Congenital, familial and genetic disorders 10080960 Hereditary angioedema type II LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
    EudraCT Number: 2015-003290-15 Sponsor Protocol Number: GTI1503 Start Date*: 2016-05-12
    Sponsor Name:Grifols Therapeutics Inc.
    Full Title: A Multi-Centre, Open-Label, Single Arm Trial to Evaluate Efficacy, Pharmacokinetics, and Safety and Tolerability of IGSC 20% in Subjects with Primary Immunodeficiency
    Medical condition: Primary Immunodeficiency (PI) diseases
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021428 - Immune system disorders 10061598 Immunodeficiency PT
    20.0 10021428 - Immune system disorders 10045792 Unspecified disorder of immune mechanism LLT
    20.0 10010331 - Congenital, familial and genetic disorders 10064859 Primary immunodeficiency syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) CZ (Completed) ES (Completed) DE (Completed) PL (Completed) HU (Completed) FR (Completed)
    Trial results: View results
    EudraCT Number: 2011-005015-82 Sponsor Protocol Number: NGAM-05 Start Date*: 2012-01-10
    Sponsor Name:Octapharma AG
    Full Title: Clinical study to evaluate the safety and tolerability of immunoglobulin intravenous (human) 10% (NewGam) administered at high infusion rates to patients with primary immunodeficiency diseases (ext...
    Medical condition: primary immunodeficiency diseases
    Disease: Version SOC Term Classification Code Term Level
    14.1 10010331 - Congenital, familial and genetic disorders 10049485 Bruton's agammaglobulinemia LLT
    14.1 10021428 - Immune system disorders 10010112 Common variable immunodeficiency LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2013-000961-36 Sponsor Protocol Number: KB057 Start Date*: 2013-11-15
    Sponsor Name:Kedrion SpA
    Full Title: Tolerability and safety evaluation of the administration of Ig VENA at high infusion rates. Open label phase III study.
    Medical condition: Patients affected by primary or secondary immunodeficiency (ID) or patients affected by Primary Immune Thrombocytopenia (ITP)
    Disease: Version SOC Term Classification Code Term Level
    16.1 10021428 - Immune system disorders 10054979 Secondary immunodeficiency PT
    16.1 10010331 - Congenital, familial and genetic disorders 10064859 Primary immunodeficiency syndrome PT
    16.1 10005329 - Blood and lymphatic system disorders 10021245 Idiopathic thrombocytopenic purpura PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DE (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2014-003772-23 Sponsor Protocol Number: ZLB05_006CR Start Date*: 2015-04-14
    Sponsor Name:CSL Behring AG
    Full Title: A Multicenter Extension Study on the Safety and Efficacy of IgPro10 in Patients With Primary Immunodeficiency (PID)
    Medical condition: Primary Immune Deficiency (Common Variable Immunodeficiency and X-linked agammaglobulinemia)
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10064859 Primary immunodeficiency syndrome PT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2015-003369-27 Sponsor Protocol Number: REGAIN Start Date*: 2016-07-01
    Sponsor Name:Universitätsklinikum Freiburg
    Full Title: A Phase I-IIa trial on low-dose IL-2 (Aldesleukin) treatment for immunological dysregulation in common variable immunodeficiency (CVID)
    Medical condition: autoimmune enteropathy (AIE) in common variable immunodeficiency (CVID).
    Disease: Version SOC Term Classification Code Term Level
    19.0 10021428 - Immune system disorders 10021449 Immunodeficiency common variable PT
    19.0 100000004856 10017922 Gastroenteropathy NOS LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed)
    Trial results: View results
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    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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