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Clinical trials for Blood and lymphatic 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
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    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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    29 result(s) found for: Blood and lymphatic system disorders congenital. Displaying page 1 of 2.
    1  2  Next»
    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: 2009-017978-21 Sponsor Protocol Number: EMR700568-012 Start Date*: 2010-12-20
    Sponsor Name:Merck Serono S.A. - Geneva
    Full Title: Prospective observational long-term safety registry of Multiple Sclerosis patients who have participated in cladribine clinical trials (PREMIERE)
    Medical condition: Multiple Sclerosis patients who have participated in cladribine tablets clinical trials
    Disease: Version SOC Term Classification Code Term Level
    20.1 10029205 - Nervous system disorders 10028245 Multiple sclerosis PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Completed) FI (Completed) LT (Completed) AT (Completed) GR (Completed) CZ (Completed) GB (Completed) EE (Completed) LV (Completed) BE (Completed) SE (Completed) PT (Completed) DK (Completed) ES (Completed) IT (Completed) PL (Completed) BG (Completed)
    Trial results: View results
    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: 2011-002052-14 Sponsor Protocol Number: EURO-HIT-HLH Start Date*: 2011-06-07
    Sponsor Name:AZIENDA OSPEDALIERA MEYER
    Full Title: EURO-HIT-HLH European cooperative pilot study for testing Hybrid ImmunoTherapy for Hemophagocytic LymphoHistiocytosis
    Medical condition: Familial hemophagoytic lymphohistiocytosis
    Disease: Version SOC Term Classification Code Term Level
    14.1 10005329 - Blood and lymphatic system disorders 10005329 Blood and lymphatic system disorders SOC
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Prematurely Ended) DE (Completed) AT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-002122-36 Sponsor Protocol Number: 64007957MMY1001 Start Date*: 2017-12-10
    Sponsor Name:Janssen-Cilag International NV
    Full Title: A Phase 1/2, First-in-Human, Open-Label, Dose Escalation Study of Teclistamab, a Humanized BCMA x CD3 Bispecific Antibody, in Subjects with Relapsed or Refractory Multiple Myeloma
    Medical condition: Relapsed or Refractory Multiple Myeloma
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10005330 Blood and lymphatic system disorders congenital HLGT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Trial now transitioned) NL (Trial now transitioned) BE (Trial now transitioned) GB (GB - no longer in EU/EEA) DE (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2011-003072-37 Sponsor Protocol Number: 8HA01EXT Start Date*: 2012-01-18
    Sponsor Name:Biogen Idec Research Ltd
    Full Title: An Open-Label, Multicenter Evaluation of the Long-Term Safety and Efficacy of Recombinant Human Coagulation Factor VIII Fusion Protein (rFVIIIFc) in the Prevention and Treatment of Bleeding Episode...
    Medical condition: Hemophilia A
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10053753 Hemophilia A without inhibitors LLT
    19.0 100000004850 10060612 Hemophilia A LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male
    Trial protocol: SE (Completed) BE (Completed) GB (Completed) DE (Completed) AT (Completed) ES (Completed) IT (Completed) IE (Completed) PL (Completed) NL (Completed)
    Trial results: View results
    EudraCT Number: 2017-002400-26 Sponsor Protocol Number: 64407564MMY1001 Start Date*: 2019-02-06
    Sponsor Name:Janssen-Cilag International NV
    Full Title: A Phase 1/2, First-in-Human, Open-Label, Dose Escalation Study of Talquetamab, a Humanized GPRC5D x CD3 Bispecific Antibody, in Subjects with Relapsed or Refractory Multiple Myeloma
    Medical condition: Relapsed or Refractory Multiple Myeloma
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10005330 Blood and lymphatic system disorders congenital HLGT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Trial now transitioned) DE (Trial now transitioned) PL (Trial now transitioned) FR (Trial now transitioned) BE (Trial now transitioned)
    Trial results: (No results available)
    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: 2018-001469-18 Sponsor Protocol Number: CT2-04-17 Start Date*: 2019-04-17
    Sponsor Name:Dipartimento di Scienze della Vita e Biotecnologie dell'Università degli Studi di Ferrara
    Full Title: Treatment of beta-thalassemia patients with rapamycin (sirolimus): from pre-clinical research to a clinical trial
    Medical condition: Beta-thalassemia transfusion dependent patients
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2007-000284-21 Sponsor Protocol Number: 524E-CVD-9101-004 Start Date*: 2007-11-08
    Sponsor Name:York Hospitals NHS Foundation Trust [...]
    1. York Hospitals NHS Foundation Trust
    2. University of York
    Full Title: A multicentre, multinational randomised control trial of prophylactic low molecular weight heparin(LMWH) in high risk pregnant thrombophilic women
    Medical condition: Thrombophilia in pregnancy. This is a multi-centre, multi-national randomised controlled trial of Low Molecular Weight Heparin to prevent pregnancy complications in high-risk pregnant thrombophi...
    Disease: Version SOC Term Classification Code Term Level
    14.0 10005329 - Blood and lymphatic system disorders 10057396 Thrombophilia LLT
    Population Age: Adults Gender: Female
    Trial protocol: GB (Completed)
    Trial results: View results
    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: 2013-000261-36 Sponsor Protocol Number: FGTW-1004 Start Date*: 2013-11-29
    Sponsor Name:LFB Biotechnologies
    Full Title: Clinical pharmacology, efficacy and safety study of FGTW in paediatric patients with severe congenital fibrinogen deficiency
    Medical condition: Afibrinogenaemia or severe hypofibrinogenaemia
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004850 10052651 Afibrinogenaemia LLT
    16.1 10005329 - Blood and lymphatic system disorders 10051125 Hypofibrinogenaemia PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: FR (Completed) 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: 2016-001223-31 Sponsor Protocol Number: KAISII Start Date*: 2016-10-11
    Sponsor Name:King's College Hospital NHS Foundation Trust
    Full Title: Incidence of Invasive Fungal Disease in Patients receiving Immunosuppressive Therapy, Intensive Chemotherapy or Reduced Intensity Haematopoietic Stem Cell Transplantation on Posaconazole Prophylaxis
    Medical condition: Aplastic Anaemia, Myelodysplastic syndromes, Acute Myeloid Leukaemia undergoing immunosuppression therapy, high dose chemotherapy or reduced intensity stem cell transplantation
    Disease: Version SOC Term Classification Code Term Level
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10038271 Refractory anaemia with excess blasts in transformation LLT
    19.0 10042613 - Surgical and medical procedures 10059041 Allogeneic peripheral haematopoietic stem cell transplant LLT
    19.0 10042613 - Surgical and medical procedures 10067862 Allogeneic stem cell transplantation LLT
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10038270 Refractory anaemia with an excess of blasts PT
    19.0 10005329 - Blood and lymphatic system disorders 10010776 Constitutional aplastic anaemia LLT
    19.0 10005329 - Blood and lymphatic system disorders 10068063 Aplastic anaemia relapse LLT
    19.0 10005329 - Blood and lymphatic system disorders 10036699 Primary idiopathic aplastic anaemia LLT
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10000880 Acute myeloid leukaemia PT
    19.0 10042613 - Surgical and medical procedures 10001756 Allogenic bone marrow transplantation therapy PT
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10000884 Acute myeloid leukaemia NOS LLT
    19.0 10005329 - Blood and lymphatic system disorders 10032510 Other specified aplastic anaemias LLT
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10066355 Treatment related acute myeloid leukaemia LLT
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10060355 Acute myeloid leukaemia in remission LLT
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10028532 Myelodysplasia LLT
    19.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10066764 Acute myeloid leukaemia progression LLT
    19.0 10005329 - Blood and lymphatic system disorders 10002967 Aplastic anaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2017-000653-39 Sponsor Protocol Number: IMR-SCD-102 Start Date*: 2017-10-19
    Sponsor Name:Imara, Inc
    Full Title: A Phase 2a, Randomised, Double-Blind, Placebo-Controlled Study of IMR-687 in Adult Patients with Sickle Cell Anaemia (Homozygous HbSS or Sickle-β0 Thalassemia)
    Medical condition: Sickle cell disease (SCD) is a rare inherited red blood cell (RBC) disorder characterized by chronic hemolysis, debilitating pain, progressive multiorgan damage, and premature death. SCD is caused ...
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA)
    Trial results: View results
    EudraCT Number: 2015-003166-91 Sponsor Protocol Number: CETB115E2201 Start Date*: 2019-05-22
    Sponsor Name:Novartis Pharma AG
    Full Title: A phase II, open-label, non-controlled, intra-patient dose escalation study to characterize the pharmacokinetics after oral administration of eltrombopag in pediatric patients with refractory, rela...
    Medical condition: Refractory, relapsed or treatment naïve severe aplastic anemia (SAA) or recurrent aplastic anemia (AA)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10005329 - Blood and lymphatic system disorders 10002967 Aplastic anaemia PT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) PT (Completed) NL (Ongoing)
    Trial results: View results
    EudraCT Number: 2021-001674-34 Sponsor Protocol Number: AG348-C-020 Start Date*: 2022-02-17
    Sponsor Name:AGIOS PHARMACEUTICALS, INC.
    Full Title: A Phase 2/3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Mitapivat in Subjects With Sickle Cell Disease.
    Medical condition: Sickle Cell Disease
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) FR (Trial now transitioned) BE (Trial now transitioned) DE (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-004471-39 Sponsor Protocol Number: IMR-SCD-301 Start Date*: 2020-06-24
    Sponsor Name:IMARA, Inc.
    Full Title: A Phase 2b Study to Evaluate the Safety and Efficacy of IMR-687 in Subjects with Sickle Cell Disease
    Medical condition: Sickle Cell Disease
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) NL (Prematurely Ended) GR (Prematurely Ended) IT (Prematurely Ended)
    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: 2020-004839-25 Sponsor Protocol Number: BO42451 Start Date*: 2021-08-18
    Sponsor Name:F. HOFFMANN - LA ROCHE LTD.
    Full Title: A RANDOMIZED DOUBLE-BLIND PHASE IIA STUDY EVALUATING THE EFFICACY, SAFETY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF CROVALIMAB AS ADJUNCT TREATMENT IN PREVENTION OF VASO-OCCLUSIVE EPISODES (VOE) I...
    Medical condition: Sickle Cell Disease (SCD); vaso-occlusive episodes in SCD
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040644 Sickle cell disease PT
    20.0 100000004851 10072397 Vaso-occlusive crisis LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) ES (Ongoing) FR (Trial now transitioned) NL (Trial now transitioned)
    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
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