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Clinical trials for Beta 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   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).

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    58 result(s) found for: Beta thalassemia. Displaying page 1 of 3.
    1  2  3  Next»
    EudraCT Number: 2009-014136-37 Sponsor Protocol Number: Thal-002 Start Date*: 2010-07-13
    Sponsor Name:University of Washington
    Full Title: A pilot study on the safety and efficacy of haemopoietic stem cell mobilization (CD34+ cells) with MOZOBIL ± G-CSF, in adult patients diagnosed with beta-thalassaemia major.
    Medical condition: Beta-thalassaemia major
    Disease: Version SOC Term Classification Code Term Level
    12.0 10004505 Beta thalassaemia LLT
    12.0 10004514 Beta-thalassaemia LLT
    12.0 10043391 Thalassaemia beta LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: GR (Completed)
    Trial results: (No results available)
    EudraCT Number: 2018-001469-18 Sponsor Protocol Number: CT2-04-17 Start Date*: 2019-04-17
    Sponsor Name:Dipartimento di Scienze della Vita e Biotecnologie dell'Università degli Studi di Ferrara
    Full Title: Treatment of beta-thalassemia patients with rapamycin (sirolimus): from pre-clinical research to a clinical trial
    Medical condition: Beta-thalassemia transfusion dependent patients
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    EudraCT Number: 2009-014666-25 Sponsor Protocol Number: 2009-014666-25 Start Date*: 2010-01-02
    Sponsor Name:FONDAZIONE FRANCO E PIERA CUTINO
    Full Title: SEQUENTIAL DEFERASIROX-DEFERIPRONE VERSUS DEFERASIROX OR DEFERIPRONE MULTICENTRE RANDOMIZED TRIAL
    Medical condition: BETA-THALASSEMIA MAJOR
    Disease: Version SOC Term Classification Code Term Level
    12.0 10004514 Beta-thalassaemia LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
    EudraCT Number: 2004-002173-23 Sponsor Protocol Number: GD3-134-201 Start Date*: 2004-11-09
    Sponsor Name:GENZYME
    Full Title: PHASE 2 CLINICAL STUDY PROTOCOL: SAFETY AND EFFICACY OF RISING DOSES OF DEFERITRIN IN BETA-THALASSEMIA PATIENTS
    Medical condition: Patients with iron overload secondary to beta-thalassemia
    Disease: Version SOC Term Classification Code Term Level
    10043391 PT
    Population Age: Adults, Elderly 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: 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: 2016-003611-35 Sponsor Protocol Number: HGB-212 Start Date*: 2017-07-24
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, by Transplantation of Autologous CD34+ Stem Cells Transduced Ex V...
    Medical condition: This study will enroll subjects with transfusion dependent beta-thalassemia, defined by a history of at least 100 mL/kg/year of packed red blood cells (pRBCs) or ≥ 8 transfusions of pRBCs per year...
    Disease: Version SOC Term Classification Code Term Level
    20.1 100000004850 10054660 Thalassemia beta LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (GB - no longer in EU/EEA) DE (Completed) GR (Completed) FR (Completed) IT (Completed)
    Trial results: View results
    EudraCT Number: 2011-000973-30 Sponsor Protocol Number: AMD-THAL Start Date*: 2011-04-28
    Sponsor Name:FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
    Full Title: PLERIXAFOR MOBILIZED STEM CELLS AS SOURCE FOR GENE THERAPY OF BETA-THALASSEMIA AMD-THAL .
    Medical condition: transfusion dependent beta thalassemia
    Disease: Version SOC Term Classification Code Term Level
    14.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: View results
    EudraCT Number: 2018-001942-33 Sponsor Protocol Number: CT2-02-17 Start Date*: 2019-01-23
    Sponsor Name:Rare Partners s.r.l. Impresa Sociale
    Full Title: A personalized medicine approach for beta-thalassemia transfusion dependent patients: testing SIROLIMUS in a first pilot clinical trial.
    Medical condition: beta-thalassemic patients
    Disease: Version SOC Term Classification Code Term Level
    20.0 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    Trial results: (No results available)
    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: 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: 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: 2015-004122-33 Sponsor Protocol Number: HGB-207 Start Date*: 2016-11-10
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase 3 Single Arm Study Evaluating the Efficacy and Safety of Gene Therapy in Subjects with Transfusion-dependent β-Thalassemia, who do not have β0/β0 Genotype, by Transplantation of Autologous ...
    Medical condition: This study will enroll subjects with transfusion dependent beta-thalassemia, who do not have beta0/beta0 genotype, defined by a history of at least 100 mL/kg/year of packed red blood cells (pRBCs) ...
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10054660 Thalassemia beta LLT
    Population Age: Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed) DE (Completed) GR (Completed) GB (GB - no longer in EU/EEA) FR (Completed)
    Trial results: View results
    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: 2015-003535-35 Sponsor Protocol Number: CICL670A0106E1 Start Date*: 2016-03-08
    Sponsor Name:Novartis Pharma AG
    Full Title: A 4-year Extension to a Phase II a Multicenter Study Evaluating Long-term Safety, Tolerability, Pharmacokinetics and Effects on Liver Iron Concentration of Repeated Doses of 10 mg/kg/Day of Deferas...
    Medical condition:
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10054661 Thalassemia major LLT
    19.0 100000004850 10054660 Thalassemia beta LLT
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2016-002959-17 Sponsor Protocol Number: CR-BD-001 Start Date*: 2016-12-02
    Sponsor Name:Kiadis Pharma Netherlands B.V.
    Full Title: An exploratory, open-label study to evaluate the safety and feasibility of ATIR201, a T-lymphocyte enriched leukocyte preparation depleted ex vivo of host alloreactive T-cells (using photodynamic t...
    Medical condition: Patients with beta-thalassemia major who are eligible for a haploidentical HSCT
    Disease: Version SOC Term Classification Code Term Level
    19.0 100000004850 10054661 Thalassemia major LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: GB (Prematurely Ended)
    Trial results: View results
    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: 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: 2017-001366-14 Sponsor Protocol Number: 207757 Start Date*: 2017-09-14
    Sponsor Name:GLAXOSMITHKLINE RESEARCH AND DEVELOPMENT
    Full Title: A long-term safety and efficacy follow-on study in participants with transfusion dependent β-thalassemia who have previously received GSK2696277 (autologous hematopoietic stem cells genetically mod...
    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, Adults Gender: Male, Female
    Trial protocol: IT (Prematurely Ended)
    Trial results: (No results available)
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