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Clinical trials for Beta cells

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   44335   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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    283 result(s) found for: Beta cells. Displaying page 1 of 15.
    1  2  3  4  5  6  7  8  9  Next» Last»»
    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: 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: 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: 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: 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: 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: 2014-004860-39 Sponsor Protocol Number: TIGET-BTHAL Start Date*: 2015-03-04
    Sponsor Name:OSPEDALE SAN RAFFAELE
    Full Title: A phase I/II study evaluating safety and efficacy of autologous hematopoietic stem cells genetically modified with GLOBE lentiviral vector encoding for the human beta-globin gene for the treatment ...
    Medical condition: Beta talassemia trasfusione dipendente
    Disease: Version SOC Term Classification Code Term Level
    17.1 10010331 - Congenital, familial and genetic disorders 10043391 Thalassaemia beta PT
    Population Age: Children, Adolescents, Under 18, Adults Gender: Male, Female
    Trial protocol: IT (Completed)
    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: 2009-013284-19 Sponsor Protocol Number: HINABS-ITI Start Date*: 2009-12-10
    Sponsor Name:Medical University Innsbruck
    Full Title: Immune tolerance induction in MS patients with neutralizing antibodies against interferon-beta
    Medical condition: multiple sclerosis patients with neutralizing antibodies against interferon-beta
    Disease:
    Population Age: Adults, Elderly Gender: Male, Female
    Trial protocol: AT (Completed)
    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: 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: 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)
    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)
    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: 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: 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: 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: 2016-004779-39 Sponsor Protocol Number: NH19708 Start Date*: 2018-02-26
    Sponsor Name:Roche Farma S.A(Soc unipersonal)que realiza el ensayo en España y que actua como representante F.Hoffmann-La Roche LTD.
    Full Title: AN OPEN-LABEL, SINGLE-ARM, MULTICENTER STUDY TO ASCERTAIN THE OPTIMAL STARTING DOSE OF MIRCERA® GIVEN SUBCUTANEOUSLY FOR THE MAINTENANCE TREATMENT OF ANEMIA IN PEDIATRIC PATIENTS WITH CHRONIC KIDNE...
    Medical condition: Chronic renal anemia
    Disease: Version SOC Term Classification Code Term Level
    20.0 100000004851 10058132 Renal anemia LLT
    20.0 100000004851 10072870 Chronic anemia LLT
    Population Age: Infants and toddlers, Children, Adolescents, Under 18 Gender: Male, Female
    Trial protocol: ES (Completed) LT (Completed) HU (Completed) FR (Completed) PL (Completed) IT (Completed)
    Trial results: View results
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