LONDON, England and PHILADELPHIA, Pennsylvania, February 14 /PRNewswire/ -- GlaxoSmithKline plc (GSK) announced today the commencement of a worldwide clinical trial to investigate the safety and efficacy of eltrombopag (SB-497115-GR) as a once-daily orally-administered drug for adult patients with previously-treated idiopathic thrombocytopenic purpura (ITP). ITP is a disorder characterized by low platelet counts leaving patients at risk of episodes of spontaneous bruising, mucosal bleeding, and in severe cases intracranial hemorrhage.
The multicenter Phase III study, has a double-blind, randomized, placebo-controlled, parallel group design. Subjects will be randomized to receive eltrombopag or placebo once daily for six weeks. The study population will comprise adults with ITP, diagnosed at least six months prior to screening, who have platelet counts <30,000/microL and who have not responded to previous treatment or have relapsed within three months of previous treatment. Those who achieve a platelet count of greater than or equal to 50,000/microL after 42 days of dosing will be considered treatment responders. Safety and quality of life outcomes also will be assessed.
"Data to date demonstrates that eltrombopag may be effective in increasing platelet counts in patients with chronic ITP and this trial will help us to further evaluate its potential," said Paolo Paoletti, MD, Senior Vice President, Oncology Medicine Development Centre, GSK. "The initiation of this trial also represents a significant milestone for GSK oncology and supportive care and underscores our continued commitment to delivering novel treatments to patients with significant unmet medical need."
Trial sites are in 33 countries, including Italy, Denmark, Poland, France, Germany, United Kingdom, Spain, Switzerland, Russia, Netherlands, Hong Kong, Pakistan, Mexico, Australia, United States, and Canada.
Top line results of the Phase II trial in ITP - which had a similar design as the Phase III trial, but tested various dosing levels - were reported in December, 2005, in conjunction with the 47th Annual Meeting of the American Society of Hematology in Atlanta. The study of 97 patients found that eltrombopag, at doses of 75 mg and 50 mg, compared with placebo (p<0.001), was successful in increasing platelet count from a baseline of less than 30,000/microL to at least 50,000/microL after 42 days of dosing. The 30 mg dose showed limited change from baseline. Further, there were no safety or tolerability events that would preclude advancement to Phase III studies.
About Eltrombopag
Eltrombopag is an investigational small-molecule thrombopoietin receptor agonist that is thought to stimulate the proliferation and differentiation of megakaryocytes, the bone marrow cells that give rise to blood platelets, and thus is considered a platelet growth factor. Because it is a small molecule, the drug is administered orally as a tablet and has less potential than large protein molecules for causing an immune system reaction. Eltrombopag was discovered as a result of a research collaboration between GlaxoSmithKline and Ligand Pharmaceuticals. It is being developed by GlaxoSmithKline.
About Thrombocytopenia
A reduction in platelet count is the defining characteristic of any type of thrombocytopenia and diagnosis can be confirmed following a routine blood test. Idiopathic thrombocytopenic purpura is an autoimmune disorder that is marked by platelet destruction and/or inadequate platelet production. The onset of symptoms is sometimes unpredictable and patients suffering from thrombocytopenia may be asymptomatic. Thrombocytopenia also can occur as a consequence of chemotherapy treatment, interferon treatment, or chronic liver disease. The prevalence of the disease is not well-established, as there are very few published epidemiologic studies of this disease in the United States. Two epidemiologic studies of incident ITP in Europe found that incidence ranged from 16 to 23 per 1 million persons per year. An internal study of a managed-care population in the United States estimated the prevalence of chronic ITP at around 77,000 cases.
Thrombocytopenia can impede a variety of medical treatments. It can prevent cancer patients from receiving their full dose of chemotherapy, prevent patients with hepatitis C infection from receiving interferon therapy or lead to dose reductions or discontinuation, and it can prevent or complicate procedures (surgical, dental, etc.) in patients with chronic liver disease and ITP.
About GlaxoSmithKline
GlaxoSmithKline is one of the world's leading research-based pharmaceutical and healthcare companies. GlaxoSmithKline is committed to improving the quality of human life by enabling people to do more, feel better and live longer. For company information visit www.gsk.com.
Information about trial enrollment can be obtained by visiting www.itpstudy.com or www.clinicaltrials.gov
Cautionary statement regarding forward-looking statements
Under the safe harbor provisions of the US Private Securities Litigation Reform Act of 1995, the company cautions investors that any forward-looking statements or projections made by the company, including those made in this Announcement, are subject to risks and uncertainties that may cause actual results to differ materially from those projected. Factors that may affect the Group's operations are described under 'Risk Factors' in the 'Operating and Financial Review and Prospects' in the company's Annual Report 2004.
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