Trial purpose
Cancer treatment
Tumor type
Haematological
Age
18 - 75
Clinical summary
Summary
The aim of this study is to assess the effectiveness and safety of using Eltrombopag in patients with early refractory ITP. Early refractory ITP is when the ITP is not responding to standard therapy within 6 months of diagnosis. Eltrombopag is approved in Australia to treat severe chronic ITP however it is not approved for use in early refractory ITP. Personal experience provided by doctors who have used this medication indicate that as well as being effective in treating patients with chronic severe ITP, it might also be effective when treating patients in the earlier stages of ITP when initial steroid therapy is no longer adequately working. This is an open label study, all patients will receive an oral tablet dose daily of eltrombopag based on their platelet count and ethnicity (patients from Japan have been shown to have an increased eltrombopag serum level by approximately 80% compared to Caucasian patients). All patients will receive the study drug for 12 weeks and assessed for bleeding episodes and platelet count. Patients deemed to have responded will stay on eltrombopag with the opportunity to dose reduce to the lowest possible dose whilst maintaining a response over time. The study will run for 130 weeks (2 1/2 years) for those who continue to respond and there will be availability of the medication to patients beyond that. Overall response will be assessed at week 12 and defined as the achievement of CR, PR and MR. Time to response and duration of response and alternative treatment free survival will also be assessed. Therapeutic response will be assessed at week 26.
Conditions
This trial is treating people with Essential Thrombocytosis
Eligibility
Inclusion
ALL of the following criteria must be met to be eligible:
1. Documented diagnosis of ITP (by exclusion) according to the ASH guidelines,
2. Age greater than or equal to 18 years,
3. Primary refractory ITP with a platelet count less than 30x109/L despite an average daily dose of at least 1mg/kg (or 75mg in patients greater than 75kg) prednisolone for at least 2 weeks OR
Recurrent ITP after an initial response to steroids which requires 10mg or more of prednisolone per day and, or, recurrent doses of IVIG to maintain a platelet count of 30x109/L or greater (within 6 months of diagnosis, noting that above this threshold, steroid toxicity with prolonged therapy is unacceptable).
4. Failure of prior splenectomy will NOT be an inclusion criteria, noting that (a) splenectomy will not be appropriate in some patients due to surgical risk and (b) particularly in younger patients, avoiding splenectomy may be desirable if the natural history of acute ITP in a subset is ultimately to resolve
Patients with ITP fulfilling the above criteria in the setting of HIV with CD4 count greater than 0.5x109/L undetectable viral load are eligible, as will be patients with secondary causes of ITP such as auto-immune disorders, lymphoproliferative disease and hepatitis C, subject to the exclusion criteria below.
Exclusion
Patients presenting with any of the following will be excluded from the study:
1. Failure or inability to provide informed consent.
2. Geographic inaccessibility prohibiting follow-up.
3. Treatment with rituximab within 8 weeks prior to consent.
4. Predicted survival of less than 12 months.
5. Patients with multisystem autoimmune disease, lymphoproliferative disorders or hepatitis C anticipated who receive disease specific therapy within the first 12 weeks (e.g., chemotherapy, cyclophosphamide, anti-viral therapy)
6. Drug-induced thrombocytopenia.
7. Known hypersensitivity to thrombopoietin Receptor agonists.
8. Pregnant or breast-feeding.
9. Reproductive potential but not willing to adhere to adequate contraception from screening and for one year after first dose of Eltrombopag.
10. HIV with CD4 count less than 500 and detectable viral load.
11. Symptomatic clinical significant arterial or venous thrombosis within 6 weeks prior to consent. Note that patients requiring anti-platelet or anti-thrombotic therapy for an event prior to 6 weeks will be eligible, with the intention that this therapy will be resumed once the platelet count reaches an appropriate level (in the order of 50x109/L for most patients). However, patients at very high risk of thrombosis e.g., antiphospholipid syndrome with prior thromboses or multiple thrombophilia risk factors will not be eligible
12. Participation in another clinical trial with any investigational drug within 30 days prior to study screening.
Exclusion
- You have certain types of non-cancer medical conditions.
- You have had certain treatments, surgical procedures or drugs.
- You are currently being treated on a clinical trial.
Clinical trials have complex eligibility criteria, and other criteria may apply for this trial. Ask your doctor about whether this trial could be right for you.
More information
Trial Identifiers
Information on this page is partially produced from ANZCTR *. View further details about this trial on the registry via the links below:
Trial sponsor
GlaxoSmithKline
Scientific Title
A Multicentre, single arm, open label study evaluating the efficacy and safety of Eltrombopag (Revolade TM) in patients with early refractory Immune Thrombocytopenia within 6 months of diagnosis (Other ID's: U1111-1144-4732)
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