Efficacy and Safety of Romiplostim Treatment of Thrombocytopenia
Research type
Research Study
Full title
A Randomized, Double Blind, Placebo Controlled Study Evaluating the Efficacy and Safety of Romiplostim Treatment of Thrombocytopenia in Subjects with Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)
IRAS ID
4706
Contact name
David Bowen
Sponsor organisation
Amgen Inc
Eudract number
2007-007258-75
Clinicaltrials.gov Identifier
Research summary
Myelodysplastic syndromes (MDS) are a group of diseases in which the production of blood cells by the bone marrow is disrupted. In some patients with MDS, the number of blood platelets is extremely low. Platelets are a subtype of blood cells that are necessary to prevent spontaneous bleeding. Therapeutic options for MDS remain limited and are generally supportive in nature. A small percentage of patients are candidates for curative therapies such as high-dose chemotherapy with stem cell transplant. Romiplostim is the investigational product being given in this study. It is made of a protein that acts like a special protein in your body called thrombopoietin (TPO). TPO binds to the bone marrow cells in your body that make blood platelets and helps these cells to make more platelets. Romiplostim can also bind to these cells and help these cells to make more platelets. By increasing platelet counts, it is thought that responding MDS patients would have a decreased risk of bleeding and a reduction in platelet transfusions. In patients who are candidates for stem cell transplantation, reducing the number of platelet transfusions may reduce the risks of stem cell transplantation. Demonstrating these meaningful clinical benefits is the potential outcome of this study. The study is a phase 2 study which is sponsored by Amgen and taking place at various sites across Great Britain and Europe, Canada, United States and Australia. The study is composed of a 26-week placebo controlled test treatment period (romiplostim versus Placebo), a 2 to 4 week interim wash-out period, a 24-week placebo controlled extended treatment period, and a 4-week follow-up period. A placebo is a dummy treatment which looks like the real study drug but contains no active ingredients/chemicals.
REC name
Yorkshire & The Humber - Leeds West Research Ethics Committee
REC reference
08/H1307/108
Date of REC Opinion
24 Oct 2008
REC opinion
Further Information Favourable Opinion