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Clinical trials for Beta Thalassemia 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
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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
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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).

    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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    16 result(s) found for: Beta Thalassemia AND Thalassemia Major. Displaying page 1 of 1.
    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: 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: 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: 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: 2015-003535-35 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: 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: 2012-002920-33 Sponsor Protocol Number: CLI00076 Start Date*: 2012-12-10
    Sponsor Name:CERUS CORPORATION
    Full Title: A Randomized Controlled Study to Evaluate Efficacy and Safety of S-303 Treated Red Blood Cells in Subjects with Thalassemia Major Requiring Chronic RBC Transfusion
    Medical condition: Subjects with Thalassemia Major Requiring Chronic RBC Transfusion
    Disease:
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed) GB (Completed)
    Trial results: View results
    EudraCT Number: 2016-001561-88 Sponsor Protocol Number: CICL670ATRO4 Start Date*: 2016-04-14
    Sponsor Name:Novartis Pharmaceuticals
    Full Title: A Phase II, Multi-center, Single-arm, Prospective Study to Evaluate the Safety and Efficacy of Deferasirox in Beta-thalassemia Major Patients After Hematopoietic Stem Cell Transplantation
    Medical condition: -Iron overload
    Disease:
    Population Age: Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: Outside EU/EEA
    Trial results: View results
    EudraCT Number: 2012-000695-42 Sponsor Protocol Number: HGB-205 Start Date*: 2012-12-31
    Sponsor Name:bluebird bio, Inc.
    Full Title: A Phase I/II Open Label Study Evaluating the Safety and Efficacy of Gene Therapy of the β-Hemoglobinopathies (Sickle Cell Anemia and β-Thalassemia Major) by Transplantation of Autologous CD34+ Stem...
    Medical condition: Severe sickle cell anemia and transfusion-dependent beta-thalassemia major. This study will enroll patients who are eligible for an allogeneic hematopoietic stem cell transplant (HSCT) but do not...
    Disease: Version SOC Term Classification Code Term Level
    14.1 100000004850 10055579 Sickle-cell beta thalassemia LLT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: FR (Completed)
    Trial results: View results
    EudraCT Number: 2010-021062-29 Sponsor Protocol Number: CICL670A2214 Start Date*: 2010-12-21
    Sponsor Name:Novartis Pharma Services AG
    Full Title: Phase II, open-label, single-arm, multicenter study to evaluate the efficacy and safety of deferasirox in combination with deferoxamine followed by deferasirox monotherapy in patients with severe c...
    Medical condition: transfusion-dependent patients with TM, DBA or congenital sideroblastic anemia with severe cardiac iron overload
    Disease: Version SOC Term Classification Code Term Level
    14.1 10027433 - Metabolism and nutrition disorders 10065974 Chronic iron overload LLT
    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: 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: 2013-002245-11 Sponsor Protocol Number: LTF-303 Start Date*: 2013-11-12
    Sponsor Name:bluebird bio, Inc.
    Full Title: Long-term Follow-up of Subjects 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 or associated clinical study will be invited to participate in this long-term follow-up study to monitor the safety and ...
    Disease: Version SOC Term Classification Code Term Level
    17.0 100000004850 10055579 Sickle-cell beta thalassemia LLT
    Population Age: Adults Gender: Male, Female
    Trial protocol: FR (Trial now transitioned) DE (Trial now transitioned) GB (GB - no longer in EU/EEA) GR (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    EudraCT Number: 2018-002915-93 Sponsor Protocol Number: ACE-536-LTFU-001 Start Date*: 2019-06-26
    Sponsor Name:Celgene Corporation
    Full Title: A phase 3B, open-label, single-arm, rollover study to evaluate long-term safety in subjects who have participated in other Luspatercept (ACE-536) clinical trials.
    Medical condition: Prior participation on a clinical trial of luspatercept (ACE-536) in protocols eligible for participation in this study ACE-536-LTFU-001 with the following medical conditions: - myelodysplastic syn...
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004850 10074356 Non-transfusion dependent thalassemia LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10068361 MDS LLT
    20.1 100000004850 10054658 Thalassemia LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074689 Post polycythemia vera myelofibrosis LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074690 Post essential thrombocythemia myelofibrosis LLT
    21.1 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074691 Post polycythaemia vera myelofibrosis LLT
    21.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10074692 Post essential thrombocythaemia myelofibrosis LLT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10028537 Myelofibrosis PT
    20.0 10029104 - Neoplasms benign, malignant and unspecified (incl cysts and polyps) 10077161 Primary myelofibrosis PT
    20.0 100000004851 10002272 Anemia LLT
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: DE (Trial now transitioned) SE (Trial now transitioned) NL (Trial now transitioned) GB (GB - no longer in EU/EEA) FR (Trial now transitioned) GR (Trial now transitioned) ES (Ongoing) BE (Completed) BG (Trial now transitioned) IT (Trial now transitioned)
    Trial results: (No results available)
    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: 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
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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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