A Phase 2 study of Luspatercept vs Placebo in Beta Thalassemia
Research type
Research Study
Full title
A PHASE 2, DOUBLE-BLIND, RANDOMIZED, PLACEBO-CONTROLLED, MULTICENTER STUDY TO DETERMINE THE EFFICACY AND SAFETY OF LUSPATERCEPT (ACE-536) VERSUS PLACEBO IN ADULTS WITH NON TRANSFUSION DEPENDENT BETA-THALASSEMIA (The BEYOND™ Study)
IRAS ID
232094
Contact name
John Porter
Contact email
Sponsor organisation
Celgene Corporation
Eudract number
2015-003225-33
Clinicaltrials.gov Identifier
112562, IND Number
Duration of Study in the UK
1 years, 11 months, 6 days
Research summary
Beta-thalassemias are a variable group of inherited blood disorders where the production of beta globin is decreased, causing ineffective red cell production and anaemia. Other features include enlarged spleen, bony deformities and iron overload. The current treatment options include blood transfusion, iron-chelation therapy, folic acid supplements, spleen removal and in selected cases marrow stem-cell transplant. Severely affected patients require regular blood transfusion (thalassaemia major or transfusion dependent thalassaemia). Less severely affected patients are anaemic but require no transfusion or only occasional transfusion (non-transfusion dependent thalassaemias)
Luspatercept is being developed to treat several conditions including beta-thalassemias and other anaemias. Luspatercept works by increasing the production of red blood cells. Preliminary data from ongoing luspatercept trials suggest that transfusion dependent beta-thalassemias experience a 33% decrease in dependence on blood transfusion. Non-transfusion dependent patients typically experience an increase in Hb of 1-2 g/dl.
The purpose of this study is to see if people with non-transfusion dependant beta-thalassemia will experience an increase in haemoglobin, without transfusions, when taking luspatercept over a continuous 12-week interval, from Week 13 to Week 24, compared to baseline. Luspatercept safety will also be evaluated. Following a 4-week screening period participants will be randomised to the Double-blind Treatment Period (DBTP) for at least 48 weeks where they will be randomised to receive either luspatercept or the placebo. The study will then be unblinded and the participants will enter the Post-Treatment Follow-up Period (PTFP) for at least 3 years. After the study unblinding, the PTFP might be performed in the rollover study. Study treatment will be administered subcutaneously in the upper arm, thigh, and/or abdomen every 21days. Study procedures will include safety and PK blood sampling, DXA scans, ECG, MRI, echocardiography and QOL questionnaires.
150 participants will be randomised at a 2:1 ratio of luspatercept versus placebo. The sponsor is Celgene.REC name
East Midlands - Leicester South Research Ethics Committee
REC reference
17/EM/0438
Date of REC Opinion
8 Jan 2018
REC opinion
Further Information Favourable Opinion