Flag of the European Union EU Clinical Trials Register Help

Clinical trials for Anemia, Diamond-Blackfan AND Thalassemia Major

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   44338   clinical trials with a EudraCT protocol, of which   7368   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 ).  
     
    Examples: Cancer AND drug name. Pneumonia AND sponsor name.
    How to search [pdf]
    Search Tips: Under advanced search you can use filters for Country, Age Group, Gender, Trial Phase, Trial Status, Date Range, Rare Diseases and Orphan Designation. For these items you should use the filters and not add them to your search terms in the text field.
    Advanced Search: Search tools
     
    3 result(s) found for: Anemia, Diamond-Blackfan AND Thalassemia Major. Displaying page 1 of 1.
    EudraCT Number: 2007-000766-20 Sponsor Protocol Number: CICL670A2206 Start Date*: 2008-07-17
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A multicenter, randomized, open-label phase II trial evaluating deferasirox compared with deferoxamine in patients with cardiac iron overload due to chronic blood transfusions
    Medical condition: Beta-thalassemia major (TM) or Diamond Blackfan anemia (DBA) or myelodysplastic syndromes (MDS) (low and INT-1 risk as per the IPSS for MDS) or sideroblastic anemia and myocardial iron overload as ...
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed)
    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: 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
    Download Options:
    Number of Trials to download:
    Download Content:
    Download Format:
    Note, where multi-state trials are shown in search results, selecting "Full Trial details" will download full information for each of the member states/countries involved in the trial.
    Query did not match any studies.
    For support, Contact us.
    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.

    European Medicines Agency © 1995-Fri May 23 09:17:12 CEST 2025 | Domenico Scarlattilaan 6, 1083 HS Amsterdam, The Netherlands
    EMA HMA