RACE version 2
Research type
Research Study
Full title
A prospective Randomized multicenter study comparing horse Antithymocyte globuline (hATG) + Cyclosporine A (CsA) with or without Eltrombopag as front-line therapy for severe aplastic anemia patients.
IRAS ID
163191
Contact name
Evelien de Ruiter
Contact email
Sponsor organisation
EBMT
Eudract number
2014-000363-40
Clinicaltrials.gov Identifier
Duration of Study in the UK
5 years, 0 months, 1 days
Research summary
Title: Will eltrombopag added to standard immunosuppressive treatment increase the rate of early (3 months) complete response (defined as Hb >10 g/dL, ANC > 1,000/μL and Plt >100,000 μL) in untreated SAA patients?
Background:
Severe aplastic anemia (SAA)is an hematological disease of the bone marrow resulting in an impairment of the production of blood cells and a lack of all blood cells.Standard treatment, Immunesuppression therapy, for SAA is based on the association of anti-thymocyte globulin (ATG) and cyclosporine A (CsA). Despite the demonstration that this treatment may result in clinical response in about two thirds of patients, about one third of patient may relapse.
Very recently a new drug, eltrombopag, has been investigated for the treatment of SAA. Eltrombopag has to be considered a compound which stimulates the functioning of the bone marrow independently from immunesuppression therapy. In a recent study eltrombopag resulted in excellent response rate in SAA patients who were refractory to immunosuppression therapy.
Design of the study:
This study will be conducted by the Severe Aplastic Anaemia Working Party of the European Group for Blood and Marrow Transplantation in seven countries throughout Europe and a total of 31 participating hospitals. The research is funded by GlaxoSmithKline. SAA patients who are not eligible for bone marrow transplantation (no available HLA-indentical donor) are approached to take part in this study. After signing consent they will be randomized in either treatment arm A (hATG + CsA) or treatment arm B (hATG + CsA + eltrombopag). For both treatment arms, patients are administered in the hospital for 4 days (hATG treatment). After these 4 days, patients can continue their treatment with CsA and for arm B eltrombopag, at home (orally). Patients are closely monitored during 22 visits divided over a total duration of 2 years (treatment + follow up).REC name
London - Hampstead Research Ethics Committee
REC reference
15/LO/1149
Date of REC Opinion
13 Aug 2015
REC opinion
Further Information Favourable Opinion