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Clinical trials for Haemoglobinopathies

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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    8 result(s) found for: Haemoglobinopathies. 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: 2012-000658-67 Sponsor Protocol Number: DEEP-1 Start Date*: 2012-07-31
    Sponsor Name:Consorzio per Valutazioni Biologiche e Farmacologiche
    Full Title: Multi-centre, oral single dose experimental and modelling study to evaluate the pharmacokinetics of deferiprone in patients aged from 1 month to less than 6 years of age affected by transfusion-dep...
    Medical condition: chronic iron overload
    Disease: Version SOC Term Classification Code Term Level
    17.1 100000004861 10065974 Chronic iron overload LLT
    Population Age: Infants and toddlers, Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed) Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-000246-62 Sponsor Protocol Number: CICL670AAU01 Start Date*: 2016-03-07
    Sponsor Name:Novartis
    Full Title: A Study of Magnetic Resonance Imaging Assessment of Cardiac and Liver Iron Load in Patients With Haemoglobinopathies, Myelodysplastic Syndromes (MDS) or Other Anaemias Treated With Exjade® (Deferas...
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004864 10028534 Myelodysplastic syndrome NOS LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2012-000353-31 Sponsor Protocol Number: DEEP-2 Start Date*: 2012-11-29
    Sponsor Name:CONSORZIO PER VALUTAZIONI BIOLOGICHE E FARMACOLOGICHE
    Full Title: Multicentre, randomised, open label, non-inferiority active-controlled trial to evaluate the efficacy and safety of deferiprone compared to deferasirox in paediatric patients aged from 1 month to l...
    Medical condition: Chronic iron overload
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004861 10065974 Chronic iron overload LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed) Outside EU/EEA GR (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2013-002454-78 Sponsor Protocol Number: V72_62 Start Date*: 2014-02-18
    Sponsor Name:Novartis Vaccines and Diagnostics S.r.l.
    Full Title: A Phase IIIb, Open Label, Controlled, Multi-Center Study to Evaluate the Safety, Tolerability and Immunogenicity of Two Doses of Novartis Meningococcal Group B Vaccine when administered to Immunoco...
    Medical condition: Meningococcal Group B disease.
    Disease: Version SOC Term Classification Code Term Level
    16.1 10021881 - Infections and infestations 10027202 Meningitis bacterial PT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: IT (Completed) ES (Completed) PL (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
    EudraCT Number: 2019-001462-15 Sponsor Protocol Number: MC-MSC.1/aGvHD Start Date*: 2020-09-24
    Sponsor Name:medac Gesellschaft für klinische Spezialpräparate mbH
    Full Title: A Randomised, Open label, Multicentre, Phase 3 Trial of First line Treatment with Mesenchymal Stromal Cells MC0518 Versus Best Available Therapy in Adult and Adolescent Subjects with Steroid refrac...
    Medical condition: Steroid refractory Acute Graft versus host Disease
    Disease: Version SOC Term Classification Code Term Level
    27.0 10021428 - Immune system disorders 10066260 Acute graft versus host disease PT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) PL (Trial now transitioned) FR (Trial now transitioned) SE (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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