ACE-011-B-THAL-001 Amendment 1 dated 4th May 2012

• Research type

  Research Study

• Full title

  A Phase 2a, open-label, dose finding study to determine the safety and tolerability of sotatercept (ACE-011) in adults with beta - thalassemia

• IRAS ID

  111039

• Contact name

  John Porter

• Sponsor organisation

  Celgene Corporation

• Eudract number

  2011-005659-15

• ISRCTN Number

  not issued

• Research summary

  Summary of Research
  The patients are being asked to participate in the study as they have been diagnosed with beta thalassemia major, or intermedia, which is a blood disorder for which they may be currently receiving blood transfusions to treat their anaemia (a decrease in red blood cells, which may cause fatigue and tiredness) related to their disease. The main purpose of this study is to find a dose of the study drug that will be useful in reducing the need for repeated, frequent transfusions in subjects with transfusion dependent beta thalassemia major or intermedia and to increase the hemoglobin level in patients with non-transfusion dependent beta-thalassemia intermedia. However, it is unknown whether any dose of the study drug will be helpful in the treatment of beta-thalassemia major or intermedia. Other purposes of this study are to determine the amounts of study drug in the body at different times, to test whether the study drug affects red blood cells, and to determine the amounts of biomarkers in the body at different times. Biomarkers are substances in the body such as proteins that can be used to show the status of disease and/or response to the study drug. This study also includes optional testing of bone marrow aspirate and blood samples for biomarkers. Blood may be collected and will be used in biomarker research conducted by the sponsor. Biomarkes in the bone marrow aspirate will be measured to see how the chemical makeup of the bone marrow aspirate plays a role in response to the study drug. Participation in the biomarker testing is optional.

  Summary of Results
  Lay summary of study results will be provided to the Investigators to pass on to the patients in the form of plain language summary (PLS).

• REC name

  London - Central Research Ethics Committee

• REC reference

  12/LO/1328

• Date of REC Opinion

  4 Oct 2012

• REC opinion

  Further Information Favourable Opinion