A Phase 3 Study of Luspatercept versus Placebo in Beta-Thalassemia

• Research type

  Research Study

• Full title

  A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of Luspatercept (ACE-536) Versus Placebo in Adults who Require Regular Red Blood Cell Transfusions Due to Beta (B)-thalassemia

• IRAS ID

  193141

• Contact name

  John Porter

• Contact email

  j.porter@ucl.ac.uk

• Sponsor organisation

  Celgene Corporation

• Eudract number

  2015-003224-31

• Clinicaltrials.gov Identifier

  NCT02604433

• Duration of Study in the UK

  3 years, 1 months, 30 days

• Research summary

  Summary of Research
  Beta-thalassemia is an inherited blood disorder that reduces the production of haemoglobin. People with beta-thalassemia have a shortage of red blood cells (anaemia) and low levels of haemoglobin which leads to a lack of oxygen in many parts of the body causing weakness, fatigue and more serious complications. The current treatment options for beta-thalassemia include blood transfusion, iron chelation therapy, folic acid supplements, blood and marrow stem cell transplant and splenectomy.

  Luspatercept is being developed to treat a number of conditions including beta-thalassemia and anaemia. Luspatercept works by increasing the production of red blood cells. Preliminary data from ongoing trials suggest that patients with beta-thalassemia receiving luspatercept experience a 33% decrease in dependence on blood transfusion.

  The main purpose of this study is to see if people with beta-thalassemia will experience an increase in their haemoglobin, and therefore a reduction in their dependence on blood transfusions, if they take luspatercept plus best supportive care (BSC). The safety of luspatercept will be also evaluated.

  Following a 12 week screening period, 67% of eligible participants will be randomised to receive luspatercept plus BSC and 33% will receive placebo plus BSC. After a 48 week double-blind treatment period, participants will enter a long-term double-blind treatment period until all enrolled patients have completed 48 weeks of treatment, discontinued, or the study is unblinded. At that point participants assigned to luspatercept may continue treatment in an open label extension study. The total study treatment period will be up to 5 years (including the extension study).
  
  Study treatment will be administered subcutaneously in the upper arm, thigh, and/or abdomen in 3 weekly cycles. Study procedures will include safety and PK blood sampling, DXA scans, ECG, MRI, echocardiography and QOL questionnaires.

  300 participants will be enrolled worldwide; the study is sponsored by Celgene Corporation.

  Summary of Results
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• REC name

  London - Bloomsbury Research Ethics Committee

• REC reference

  16/LO/0083

• Date of REC Opinion

  14 Mar 2016

• REC opinion

  Further Information Favourable Opinion