A Phase 3, Double-blind, Randomized, Placebo-controlled, Multicenter Study to Determine the Efficacy and Safety of Luspatercept the Study Drug Versus Placebo in Adults who Require Regular Red Blood Cell Transfusions Due to Beta-Thalassemia
November 10, 2017
UCSF Benioff Children's Hospital Oakland has completed enrollment for this study, and is no longer enrolling patients.
This study is intended to test the safety and efficacy of a drug called Luspatercept for people with transfusion-dependent Beta-thalassemia. The investigators would like to know whether Luspatercept can increase hemoglobin levels and reduce the number of units of blood people receive during transfusions or increase the amount of time between transfusions.
This study may be a good fit for you if:
- You are 18 years or older
- You have diagnosis of Beta-thalassemia or Hemoglobin E/B-thalassemia and received between 6 and 20 units of blood in the past 24 weeks. (A unit is defined as 350 ml.)
If You Decide to Participate in This Study:
- You will enter a screening period of 12 weeks during which you will have some blood tests, have an MRI scan of your heart, liver, spleen and pancreas, receive and ECG and a DXA bone density scan. The number of units you are receiving during your transfusions will also be calculated during this time.
- Following screening, you have a 67% chance of being assigned to Luspatercept, and a 33% chance of being assigned to placebo.
- The study consists of a 48 week Treatment Phase and a subsequent 48 week Long Term Treatment Phase. You will receive Luspatercept every 3 weeks during both of these phases, and have MRI, DXA, and Quality of Life assessments about every 6 months.
- There is an Open Label period following the two treatment phases during which everyone will receive Luspatercept.
- Subjects receive $50 compensation and travel cost reimbursement per visit.
Contact: Jennifer Ferguson