Clinical trial update (April 2017)

Important Note: The latest Clinical Trial updates can be found in the link below.


Latest Clinical trial updates

April 2017 update

Luspatercept (ACE-536):

  • A novel agent that increases haemoglobin by promoting erythropoiesis (differentiation and maturation of late-stage erythrocyte precursors).
  • Phase 2 data in adults with β-thalassaemia shows sustained increase in haemoglobin levels with reduction of transfusion requirement and improvement in quality of life. An improvement in iron overload was also noted. The drug was well tolerated with mild to moderate side effects such muscle and joint pains.
  • A recently concluded Phase 2 trial addressed the efficacy and safety of luspatercept in adults with NTDT. In this group of patients haemoglobin levels, the liver iron was decreased and quality of life improved
  • An ongoing Phase 3 trial (BELIEVE) evaluates the efficacy and safety of luspatercept in patients with transfusion-dependent β-thalassemia. This trial started in 2016 and is ongoing in many sites across the world. It involves patients/volunteers (up to 300 will take part) who are over 18 years old, and who fulfil certain criteria, such as being free of hepatitis, or diabetes or have major organ damage. Psychiatric illness is also a reason for exclusion from the trial. It is hoped that the trials will be concluded by late 2018 and early 2109


LentiGlobin BB305 Drug Product (DP)



  • An agent that regulates hemoglobin gene expression, approved and used for the treatment of other haematological conditions, such as myelofibrosis and resistant to hydroxyurea polycythemia vera. Ruxolitinib, belongs to a group of drugs that have been shown to reduce ineffective erythropoiesis.
  • A Phase 2 trial (TRUTH) in transfusion-dependent β-thalassemia with splenomegaly, showed that ruxolitinib therapy resulted in slight improvement in pre-transfusion haemoglobin levels and survival of transfused red cells with a noticeable reduction in spleen size, rendering the drug a promising alternative to splenectomy.
    Aydinok Y, Karakas Z, Cassinerio E, et al in Blood 2016, 1`28:852





  • A calcium channel blocker that prevents entrance of iron into cells, used for the treatment of hypertension and coronary artery disease.
  • A recently published randomized trial showed that addition of amlodipine to standard iron chelation therapy in thalassemia major patients with cardiac siderosis, amlodipine combined with chelation therapy reduced cardiac iron more effectively than chelation therapy alone.
  • An ongoing randomized trial (CANALI) evaluates the efficacy and safety of adding amlodipine on top of deferasirox on cardiac iron in transfusion-dependent thalassaemia patients with moderate cardiac iron overload (MRI T2* 10-20 ms).



  • A new for the treatment of vaso-occlusive events in sickle cell disease (antibody against the adhesion molecule P-selectin)
  • A recently published Phse 2 trial showed that Crizanlizumab reduced significantly pain crises with a low incidence of adverse events. Ataga KI, Kutlar A, Kanter J, Liles D, et al N Engl J Med 2017 376(5): 429-439
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