Clinical Trials

A clinical trial in the European Union is defined by Directive 2001/20/EC, as a clinical study that aims to investigate the therapeutic effect(s) of medicinal products that have not yet been authorized by regulatory bodies such as the European Medicines Agency (EMA), or the value of authorized drugs in cases other than those for which it has been authorized. In addition, clinical trials are considered those studies that seek to assign specific products to a therapeutic strategy or propose to investigate effective diagnostic or monitoring procedures.

  • ACE-536: Phase 2 data from both the dose-escalation and the ongoing long-term extension study were presented during the 21st Congress of the European Hematology Association, 10 June 2016, in Copenhagen. The latest data show that Luspatercept (ACE-536) has sustained increases in hemoglobin levels, reduced transfusion burden, and improved patient reported quality of life measures in adults with Beta-Thalassemia.”
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  • ACE-536: Celgene in partnership with Acceleron are now enrolling patients in a Phase 3 clinical trial that is designed to evaluate the safety and efficacy of luspatercept in patients with beta-thalassemia.  The clinical trial is called the “BELIEVE” study.
    For more information, click here
  • HGB-205: New clinical data from bluebird bio gene therapy studies was released at 56th American Society of Hematology (ASH) Annual Meeting & Exposition, 7 December 2014, in San Francisco. The first four Beta thalassemia major patients treated in these studies who have at least 3 months of follow-up are now all transfusion free, support by rapid and robust production of the therapeutic globin from the gene vector. The first ever sickle cell disease patient was also treated with gene therapy (to our knowledge) in the HGB-205 study in October.
    For more information, click here
  • ACE-536: Phase 2 Study presented during the 56th American Society of Hematology (ASH) Annual Meeting & Exposition, 7 December 2014, in San Francisco. The recent data have shown that Luspatercept (ACE-536) has increased Hemoglobin and decreased the transfusion burden and serum ferritin levels in Adults with Beta-Thalassemia.
    For more information, click here
  • HGB-205: bluebird bio Reports Early Transfusion Independence in Beta-Thalassemia Major Patients Treated with its LentiGlobin® BB305 Drug Product Candidate.
    For more information, click here
  • HGB-206: A Phase I Gene Therapy Study Evaluating the Safety of the LentiGlobin® BB305 Drug Product in Severe Sickle Cell Disease Subjects (sponsored by bluebird bio):
    For more information, click here
  • HGB-204: A Phase I/II Gene Therapy Study Evaluating the Safety and Efficacy of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major Subjects (sponsored by bluebird bio).
    For more information, click here
  • HGB-205: A Phase I/II Gene Therapy Study Evaluating the Efficacy and Safety of the LentiGlobin® BB305 Drug Product in Beta-Thalassemia Major and Severe Sickle Cell Disease Subjects (sponsored by bluebird bio).
    For more information, click here
  • ACE-011: Phase 2 Trial of Sotatercept in Beta-Thalassemia at 2013 American Society of Hematology Annual Meeting.
    For more information, click here
  • The European Medicines Agency has launched an online register providing public access to information on clinical trials for medicinal products authorised in the European Union.
    For more information, click here
  • The ITHANET portal is a community website dedicated to thalassaemia and other haemoglobinopathies supplying a comprehensive list of undergoing clinical trials.
    For more information, click here
  • ACE-536: A Phase 2, Open-Label, Ascending Dose Study to Evaluate the Effects of ACE-536 in Patients With Beta-Thalassemia Intermedia.
    For more information, click here
  • ACE-011: A Phase 2a, Open-label, Dose Finding Study to Determine the Safety and Tolerability of SOTATERCEPT (ACE-011) in Adults With Beta (β)-Thalassemia.
    For more information, click here