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Clinical trials for Hemoglobinopathies

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   44336   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: Hemoglobinopathies. Displaying page 1 of 1.
    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: 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: 2008-005594-35 Sponsor Protocol Number: TJB0603 Start Date*: 2009-10-28
    Sponsor Name:CHU Sart-Tilman
    Full Title: Randomized double-blind study of mesenchymal stem cells (MSC) in patients undergoing matched unrelated allogeneic bone marrow or peripheral blood stem cell transplantation- A European multicentre s...
    Medical condition: Hematological disorders of the following types: ALL: acute lymphoblastic leukemia in complete remission (<5% blasts in marrow) up to and including third remission (excluding relapse). AML: acute my...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10027703 Mismatched donor bone marrow transplantation therapy LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2005-001290-10 Sponsor Protocol Number: CICL670A2203 including amendment 1 Start Date*: 2006-01-30
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A study to provide expanded access of EXJADE® (deferasirox) to patients with congenital disorders of red blood cells and chronic iron overload from blood transfusions who cannot adequately be treat...
    Medical condition: Chronic iron overload from blood transfusions in patients with congenital disorders of red blood cells
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) BE (Completed) IE (Prematurely Ended) ES (Completed) DE (Completed) GR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2006-006577-25 Sponsor Protocol Number: 070101 Start Date*: 2007-07-25
    Sponsor Name:Karolinska Institutet
    Full Title: A prospective randomized study comparing rapamune and tacrolimus vs. cyclosporine and methotrexate as immune prophylaxis in allogeneic hematopoietic stem cell transplantation, using HLA-A, -B, -DRB...
    Medical condition: Graft versus host disease prophylaxis in patients receiving stem cell transplantation due to: chronic myeloid leukemia (CML) in 1st or 2nd chronic phase, acute myeloid leukemia (AML) in complete re...
    Disease: Version SOC Term Classification Code Term Level
    9.1 10018799 GVHD LLT
    Population Age: Newborns, Infants and toddlers, Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: SE (Completed) FI (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: 2014-003572-23 Sponsor Protocol Number: GCP#01.01.030 Start Date*: 2017-12-27
    Sponsor Name:Gamida Cell Ltd
    Full Title: Allogeneic Stem Cell Transplantation of CordIn™, Umbilical Cord Blood-Derived Ex Vivo Expanded Stem and Progenitor Cells, in Patients with Hemoglobinopathies
    Medical condition: Hemoglobinopathies
    Disease: Version SOC Term Classification Code Term Level
    18.0 100000004850 10054658 Thalassemia LLT
    18.0 100000004850 10040645 Sickle cell disease NOS LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2022-000905-29 Sponsor Protocol Number: RENOIR Start Date*: 2022-04-12
    Sponsor Name:ISTITUTO NAZIONALE PER LE MALATTIE INFETTIVE "LAZZARO SPALLANZANI"
    Full Title: Temporal kinetics of antibody and cellular response markers and relative impact of revaccination in patients recovered from COVID-19 after treatment with monoclonal antibodies
    Medical condition: Covid-19 infection
    Disease: Version SOC Term Classification Code Term Level
    20.0 10021881 - Infections and infestations 10047438 Viral infectious disorders HLGT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2013-002245-11 Sponsor Protocol Number: LTF-303 Start Date*: 2013-11-12
    Sponsor Name:bluebird bio, Inc.
    Full Title: Long-term Follow-up of Subjects Treated with Ex Vivo Gene Therapy using Autologous Hematopoietic Stem Cells Transduced with a Lentiviral Vector
    Medical condition: Subjects treated with gene therapy drug products in a bluebird bio-sponsored or associated clinical study will be invited to participate in this long-term follow-up study to monitor the safety and ...
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10055579 Sickle-cell beta thalassemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) DE (Trial now transitioned) GB (GB - no longer in EU/EEA) GR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2012-000695-42 Sponsor Protocol Number: HGB-205 Start Date*: 2012-12-31
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase I/II Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the β-Hemoglobinopathies (Sickle Cell Anemia and β-Thalassemia Major) by Transplantation of Autologous CD34+ Stem...
    Medical condition: Severe sickle cell anemia and transfusion-dependent beta-thalassemia major. This study will enroll patients who are eligible for an allogeneic hematopoietic stem cell transplant (HSCT) but do not...
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10055579 Sickle-cell beta thalassemia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2015-004813-26 Sponsor Protocol Number: GCP#04.01.020/030 Start Date*: 2016-03-14
    Sponsor Name:Gamida Cell Ltd
    Full Title: Long Term Follow Up for Patients who have received Allogeneic Stem Cell Transplantation of NiCord®/CordIn™, Umbilical Cord Blood-derived Ex Vivo Expanded Stem and Progenitor Cells
    Medical condition: High risk haematological malignancies Hemoglobinopathies
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004864 10025316 Lymphoma NOS LLT
    18.1 100000004864 10000845 Acute lymphoblastic leukemia LLT
    18.1 100000004864 10000886 Acute myeloid leukemia LLT
    18.1 100000004850 10054658 Thalassemia LLT
    18.1 100000004864 10009015 Chronic myeloid leukemia LLT
    18.1 100000004850 10040645 Sickle cell disease NOS LLT
    18.1 100000004864 10028534 Myelodysplastic syndrome NOS LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: ES (Completed)
    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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