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Clinical trials for Hemoglobinopathy

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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    • 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   43893   clinical trials with a EudraCT protocol, of which   7300   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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    21 result(s) found for: Hemoglobinopathy. Displaying page 1 of 2.
    1  2  Next»
    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 (Ongoing)
    Trial results: View results
    EudraCT Number: 2007-004164-51 Sponsor Protocol Number: LPI-1/2007 Start Date*: 2008-03-26
    Sponsor Name:FONDAZIONE ITALIANA "LEONARDO GIAMBRONE"PER LA GUARIGIONE DALLA TALASSEMIA - ONLUS
    Full Title: LPI-Labile Plasma Iron in Deferasirox-Treated Thalassemic Patients
    Medical condition: thalassemic patients diagnosed with chronic iron overload due to regular blood transfusion
    Disease: Version SOC Term Classification Code Term Level
    9.1 10043388 Thalassaemia LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2015-003532-12 Sponsor Protocol Number: CICL670AUS04 Start Date*: 2016-03-07
    Sponsor Name:Novartis
    Full Title: An Open Label Trial Evaluating Cardiac T2* in Beta-thalassemia Patients on Deferasirox (ICL670) Treatment for 18 Months
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10054658 Thalassemia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2007-007000-15 Sponsor Protocol Number: CICL670A2209 Start Date*: 2009-01-19
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A randomized, double-blind, placebo-controlled, phase II study to evaluate efficacy and safety of deferasirox in nontransfusion-dependent thalassemia patients with iron overload (THALASSA)
    Medical condition: Chronic iron overload in patients with non-transfusion-dependent thalassemia
    Disease: Version SOC Term Classification Code Term Level
    9.1 10043389 Thalassaemic disorders HLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: GB (Completed) IT (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2013-002812-28 Sponsor Protocol Number: CINC424X2201 Start Date*: 2015-05-12
    Sponsor Name:NOVARTIS FARMA S.p.A
    Full Title: A single arm, multicenter, phase IIa study to explore the efficacy and safety of ruxolitinib (INC424) in regularly transfused patients with thalassemia
    Medical condition: Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    16.1 100000004850 10054658 Thalassemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2020-004995-17 Sponsor Protocol Number: NL7507301820 Start Date*: 2021-03-11
    Sponsor Name:AMsterdam UMC-AMC
    Full Title: Low dose iron chelation as TReatment of Oxidative stress in Sickle cell disease; TROS study
    Medical condition: Sickle cell disease (an hereditary hemoglobinopathy)
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2009-018091-34 Sponsor Protocol Number: CICL670AGR02 Start Date*: 2010-07-14
    Sponsor Name:Novartis (Hellas) S.A.C.I
    Full Title: A multicenter open label phase II study to evaluate the safety and efficacy of deferasirox in combination with deferioxamine followed by transitioning to deferasirox monotherapy in β-thalassemia pa...
    Medical condition: Chronic iron overload in patients cardiac iron overload and β thalassemia
    Disease: Version SOC Term Classification Code Term Level
    9.1 10043389 LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GR (Completed)
    Trial results: View results
    EudraCT Number: 2017-003777-34 Sponsor Protocol Number: NL63198.041.17 Start Date*: 2018-02-09
    Sponsor Name:UMC Utrecht
    Full Title: Proton pump inhibition for secondary hemochromatosis in hereditary anemia, a phase III placebo controlled randomized cross-over clinical trial.
    Medical condition: Iron overload (secondary hemochromatosis) in non-transfusion dependent forms of hereditary anemias. Iron overload and hereditary anemia are a growing, underestimated emerging health care problem.
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004851 10002272 Anemia LLT
    20.0 100000004861 10057875 Secondary haemochromatosis LLT
    20.0 100000004861 10022979 Iron excess HLT
    20.0 100000004850 10055610 Hemoglobinopathy LLT
    20.0 100000004850 10074356 Non-transfusion dependent thalassemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: NL (Completed)
    Trial results: View results
    EudraCT Number: 2015-003531-35 Sponsor Protocol Number: CICL670ARU01 Start Date*: 2016-03-07
    Sponsor Name:Novartis Pharmaceuticals
    Full Title: 1 Year, Open-label Multicenter Evaluation of Efficacy, Safety of Deferasirox in Patients MDS, Thalassemia and Rare Anemia Types Having Transfusion-induced Iron Overload.
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10054658 Thalassemia LLT
    19.0 100000004864 10028534 Myelodysplastic syndrome NOS LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2012-000955-15 Sponsor Protocol Number: 2012-101 Start Date*: 2012-10-02
    Sponsor Name:AZIENDA OSPEDALIERA POLICLINICO DI MODENA
    Full Title: A phase IIa open-label, single arm, multi-center trial evaluating the safety of standard antiviral therapy (pegylated interferon and ribavirin) plus deferasirox in thalassemia patients with transfu...
    Medical condition: Thalassemia patients with transfusion dependent iron overload and chronic hepatitis C
    Disease: Version SOC Term Classification Code Term Level
    15.0 10021881 - Infections and infestations 10008912 Chronic hepatitis C LLT
    15.0 10010331 - Congenital, familial and genetic disorders 10043388 Thalassaemia PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2016-000186-23 Sponsor Protocol Number: CICL670AIC04 Start Date*: 2016-06-21
    Sponsor Name:Novartis Pharma Services AG
    Full Title: Open-label, multicenter, single arm, phase III study to collect additional safety and efficacy data with deferasirox film-coated tablets in patients completing study CICL670F2201
    Medical condition: transfusion-dependent thalassemia or myelodysplastic syndrome at very low, low or intermediate (int-1) risk
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054658 Thalassemia LLT
    20.0 100000004864 10028534 Myelodysplastic syndrome NOS LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) GR (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: 2015-003224-31 Sponsor Protocol Number: ACE-536-B-THAL-001 Start Date*: 2016-03-14
    Sponsor Name:Celgene Corporation
    Full Title: A Phase 3, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Adults Who Require Regular Red Blood Cell...
    Medical condition: Adults who require regular Red Blood Cell (RBC) transfusions due to Beta-Thalassemia.
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054658 Thalassemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Prematurely Ended) BG (Completed) GR (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2013-004167-32 Sponsor Protocol Number: CICL670F2201 Start Date*: 2014-05-06
    Sponsor Name:Novartis Pharma Services AG
    Full Title: A randomized, open-label, multicenter, two arm, phase II study to investigate the benefits of an improved deferasirox formulation (film-coated tablet)
    Medical condition: transfusion-dependent thalassemia or myelodysplastic syndrome at very low, low or intermediate (int-1) risk
    Disease: Version SOC Term Classification Code Term Level
    18.1 100000004850 10054658 Thalassemia LLT
    18.1 100000004864 10028534 Myelodysplastic syndrome NOS LLT
    Population Age: Children, Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed) ES (Completed) IT (Completed) GB (Completed) FR (Completed) GR (Completed)
    Trial results: View results
    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: 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: 2021-005267-48 Sponsor Protocol Number: 4202-HEM-201 Start Date*: 2022-10-17
    Sponsor Name:FORMA THERAPEUTICS, INC.
    Full Title: A Phase 2 Open-Label Study to Evaluate Safety and Clinical Activity of FT-4202 in Patients with Thalassemia or Sickle Cell Disease
    Medical condition: Sickle cell disease (SCD) or thalassemia
    Disease: Version SOC Term Classification Code Term Level
    21.0 100000004850 10040646 Sickle cell disorders LLT
    20.1 100000004850 10040649 Sickle cell thalassaemia LLT
    Population Age: Adolescents, Under 18, Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Ongoing)
    Trial results: (No results available)
    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)
    EudraCT Number: 2013-002245-11 Sponsor Protocol Number: LTF-303 Start Date*: 2020-01-31
    Sponsor Name:bluebird bio, Inc.
    Full Title: Long-term Follow-up of Subjects With Transfusion-Dependent β-Thalassemia 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 study will be invited to participate in this long-term follow-up study to monitor the safety and efficacy of the drug pr...
    Disease: Version SOC Term Classification Code Term Level
    23.1 100000004850 10082045 Beta-thalassemia trait LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Ongoing) GB (GB - no longer in EU/EEA) GR (Ongoing) IT (Ongoing)
    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
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