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Clinical trials for β-thalassemia

The European Union Clinical Trials Register   allows you to search for protocol and results information on:
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    The EU Clinical Trials Register currently displays   44339   clinical trials with a EudraCT protocol, of which   7369   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).

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    40 result(s) found for: β-thalassemia. Displaying page 1 of 2.
    1  2  Next»
    EudraCT Number: 2011-005659-15 Sponsor Protocol Number: ACE-011-B-THAL-001 Start Date*: 2012-10-04
    Sponsor Name:Celgene Corporation
    Full Title: A Phase 2a, open-label, dose finding study to determine the safety and tolerability of sotatercept (ACE-011) in adults with beta (β)- thalassemia
    Medical condition: Subjects with red blood cell (RBC) transfusion dependent β-thalassemia major and β thalassemia intermedia, as well as non-transfusion dependent β-thalassemia intermedia will take part in this study.
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10062923 Thalassemia intermedia LLT
    20.1 100000004850 10054661 Thalassemia major LLT
    20.1 100000004850 10054660 Thalassemia beta LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) IT (Prematurely Ended) GR (Completed)
    Trial results: View results
    EudraCT Number: 2016-004799-23 Sponsor Protocol Number: BP39642 Start Date*: 2017-09-04
    Sponsor Name:F. HOFFMANN - LA ROCHE LTD.
    Full Title: A PHASE II, SINGLE ARM, MULTICENTER, PROOF OF MECHANISM STUDY TO INVESTIGATE THE SAFETY, TOLERABILITY, EFFICACY, PHARMACOKINETICS, AND PHARMACODYNAMICS OF BITOPERTIN (RO4917838) IN ADULTS WITH NON-...
    Medical condition: β-Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10074356 Non-transfusion dependent thalassemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2022-002395-36 Sponsor Protocol Number: P-SP420-THAL-01 Start Date*: 2022-12-16
    Sponsor Name:Pharmacosmos A/S
    Full Title: An open-label, dose-escalation, dose-finding, and proof-of-concept trial of SP-420 in subjects with transfusion-dependent β-thalassemia
    Medical condition: Transfusion-dependent β-thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.1 10010331 - Congenital, familial and genetic disorders 10054660 Thalassemia beta LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: DK (Trial now transitioned) GR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2014-001936-12 Sponsor Protocol Number: MD2014.01 Start Date*: 2019-02-11
    Sponsor Name:Sanquin Plasma Products BV
    Full Title: Efficacy and Safety of human apotransferrin in patients with β-thalassemia
    Medical condition: Beta thalassemia intermedia
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054660 Thalassemia beta LLT
    20.1 100000004850 10062923 Thalassemia intermedia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2017-003351-38 Sponsor Protocol Number: CTX001-111 Start Date*: 2018-07-25
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 1/2/3 Study of the Safety and Efficacy of a Single Dose of Autologous CRISPR-Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (hHSPCs) in Subjects with Transfusion-Dependen...
    Medical condition: Transfusion-Dependent β Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) IT (Completed) GB (Completed)
    Trial results: (No results available)
    EudraCT Number: 2013-000743-33 Sponsor Protocol Number: SPD602-204 Start Date*: 2014-01-16
    Sponsor Name:Shire Development LLC and International Affiliates
    Full Title: A 48-week, Open-label, 2-arm, Parallel-group, Randomized Exploratory Study to Assess Liver Iron Concentration Measured by FerriScan® (R2) Magnetic Resonance Imaging in β-thalassemia Subjects Admin...
    Medical condition: Patients 18 years of age and older with transfusional iron overload due to β-thalassemia
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004861 10065974 Chronic iron overload LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: BE (Completed) GB (Prematurely Ended) GR (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2012-002499-15 Sponsor Protocol Number: A536-04 Start Date*: 2012-12-19
    Sponsor Name:ACCELERON PHARMA INC
    Full Title: A Phase 2, Open-Label, Ascending Dose Study to Evaluate the Effects of ACE-536 in Patients with β-Thalassemia Intermedia
    Medical condition: β-thalassemia intermedia
    Disease: Version SOC Term Classification Code Term Level
    15.1 100000004850 10062923 Thalassemia intermedia LLT
    15.1 100000004850 10054660 Thalassemia beta LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2018-001984-21 Sponsor Protocol Number: PTG-300-02 Start Date*: 2018-12-17
    Sponsor Name:Protagonist Therapeutics, Inc.
    Full Title: Phase 2 Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-Thalassemia Subjects with Chronic Anemia
    Medical condition: β-Thalassemia Subjects with Chronic Anemia (transfusion and non transfusion dependent)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Completed) GR (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2014-001281-94 Sponsor Protocol Number: A536-06 Start Date*: 2015-03-11
    Sponsor Name:Acceleron Pharma, Inc.
    Full Title: An Open-Label Extension Study to Evaluate the Long-Term Effects of ACE-536 in Patients with β-Thalassemia Previously Enrolled in Study A536-04
    Medical condition: β-Thalassemia Intermedia and β-Thalassemia major
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10062923 Thalassemia intermedia LLT
    17.0 100000004850 10054661 Thalassemia major LLT
    17.0 100000004850 10054660 Thalassemia beta LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed) GR (Completed)
    Trial results: View results
    EudraCT Number: 2018-004423-36 Sponsor Protocol Number: PTG-300-03 Start Date*: 2019-04-08
    Sponsor Name:Protagonist Therapeutics, Inc.
    Full Title: An Open Label Extension Study of PTG-300 in Non-Transfusion Dependent (NTD) and Transfusion-Dependent (TD) β-thalassemia Subjects
    Medical condition: β-Thalassemia Subjects with Chronic Anemia (transfusion and non transfusion dependent)
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GR (Completed) GB (Completed) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2019-002989-12 Sponsor Protocol Number: IMR-BTL-201 Start Date*: 2020-06-02
    Sponsor Name:IMARA, Inc.
    Full Title: A Phase 2 Study to Evaluate the Safety and Tolerability of IMR-687 in Subjects with Beta Thalassemia
    Medical condition: β-thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054660 Thalassemia beta LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DK (Prematurely Ended) NL (Prematurely Ended) FR (Prematurely Ended) GR (Prematurely Ended) IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2015-003225-33 Sponsor Protocol Number: ACE-536-B-THAL-002 Start Date*: 2018-01-08
    Sponsor Name:Celgene Corporation
    Full Title: A Phase 2, Double-Blind, Randomized, Placebo-Controlled, Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) versus Placebo in Adults with Non Transfusion Dependent Bet...
    Medical condition: Non transfusion dependent β-thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10074356 Non-transfusion dependent thalassemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) GR (Completed) IT (Completed)
    Trial results: View results
    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: 2019-003505-96 Sponsor Protocol Number: ISIS702843-CS2 Start Date*: 2020-01-21
    Sponsor Name:Ionis Pharmaceuticals, Inc.
    Full Title: A Phase 2a, Randomized, Open-Label Study to Evaluate the Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of ISIS 702843 Administered Subcutaneously to Patients with Non-Transf...
    Medical condition: Non-Transfusion Dependent β-Thalassemia Intermedia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10074356 Non-transfusion dependent thalassemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: GR (Completed)
    Trial results: View results
    EudraCT Number: 2019-000208-13 Sponsor Protocol Number: ACE-536-B-THAL-004 Start Date*: 2019-12-09
    Sponsor Name:Celgene Corporation
    Full Title: A phase 2a study to evaluate the safety and pharmacokinetics of Luspatercept (ACE-536) in paediatric participants who require regular red blood cell transfusions due to beta (β) thalassemia
    Medical condition: Beta-Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054660 Thalassemia beta LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) GR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-002935-88 Sponsor Protocol Number: VX18-CTX001-131 Start Date*: 2019-03-13
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Long-term Follow-up Study of Subjects With β-thalassemia or Sickle Cell Disease Treated with Autologous CRISPR-Cas9 Modified Hematopoietic Stem Cells (CTX001)
    Medical condition: Transfusion-dependent β thalassemia (TDT). Severe sickle cell disease (SCD).
    Disease: Version SOC Term Classification Code Term Level
    26.1 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    21.0 10010331 - Congenital, familial and genetic disorders 10040641 Sickle cell anaemia PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) GB (GB - no longer in EU/EEA) BE (Trial now transitioned) FR (Ongoing) GR (Prematurely Ended) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-002172-39 Sponsor Protocol Number: VX21-CTX001-141 Start Date*: 2021-10-05
    Sponsor Name:VERTEX PHARMACEUTICALS INCORPORATED
    Full Title: A Phase 3 Study to Evaluate the Safety and Efficacy of a Single Dose of CTX001 in Pediatric Subjects With Transfusion-Dependent ß-Thalassemia
    Medical condition: Transfusion-Dependent ß Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054660 Thalassemia beta LLT
    Population Age: Children, Under 18 Gender: Male, Female
    Trial protocol: IT (Trial now transitioned) DE (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2021-000212-34 Sponsor Protocol Number: AG348-C-018 Start Date*: 2021-10-11
    Sponsor Name:Agios Pharmaceuticals, Inc.
    Full Title: A Phase 3, Double-blind, Randomized, Placebo-Controlled, Multicenter Study Evaluating the Efficacy and Safety of Mitapivat in Subjects With Transfusion-Dependent Alpha- or Beta-Thalassemia (ENERGIZ...
    Medical condition: Transfusion-Dependent Alpha- or Beta-Thalassemia
    Disease: Version SOC Term Classification Code Term Level
    22.0 100000004850 10081904 Transfusion dependent thalassemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: ES (Ongoing) DK (Trial now transitioned) IT (Trial now transitioned) BG (Trial now transitioned) FR (Trial now transitioned) NL (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2019-003766-41 Sponsor Protocol Number: COVERAGE Start Date*: 2020-09-16
    Sponsor Name:Amsterdam UMC-AMC
    Full Title: The effect of Voxelotor on Cerebral Perfusion and Oxygenation (Coverage Study)
    Medical condition: Sickle Cell Disease
    Disease:
    Population Age: Adults Gender: Male, Female
    Trial protocol: NL (Ongoing)
    Trial results: (No results available)
    EudraCT Number: 2021-006390-37 Sponsor Protocol Number: VX21-CTX001-161 Start Date*: Information not available in EudraCT
    Sponsor Name:Vertex Pharmaceuticals Incorporated
    Full Title: A Phase 3b Study to Evaluate Efficacy and Safety of a Single Dose of Autologous CRISPR Cas9 Modified CD34+ Human Hematopoietic Stem and Progenitor Cells (CTX001) in Subjects with Transfusion-Depend...
    Medical condition: Transfusion-dependent β-thalassemia (TDT) and severe sickle cell disease (SCD)
    Disease: Version SOC Term Classification Code Term Level
    21.0 10010331 - Congenital, familial and genetic disorders 10040641 Sickle cell anaemia PT
    20.1 10010331 - Congenital, familial and genetic disorders 10054660 Thalassemia beta LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
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