Study in Adult Subjects with Primary Immune Thrombocytopenia (ITP)
Research type
Research Study
Full title
Open Label, Adaptive Design, Ascending, Multiple-Dose Study to Evaluate Safety and Efficacy of BMS-986004 (Anti-CD40L dAb) in Adult Subjects with Primary Immune Thrombocytopenia (ITP)
IRAS ID
164135
Contact name
Drew Provan
Contact email
Sponsor organisation
Bristol Myers Squibb International Corporation
Eudract number
2014-001429-33
Duration of Study in the UK
2 years, 0 months, 29 days
Research summary
This clinical trial will be looking at a new study treatment currently known as BMS 986004, which is an antibody designed to target the cells which are responsible for the autoimmune response associated with Primary Immune Thrombocytopenia. (ITP). BMS-945429 may help block a part of the body’s immune system and stop the body from destroying its own platelets.
Patients with ITP are initially treated with steroids and intravenous immunoglobulin to increase their platelet count to a safe level. Some patients then maintain an adequate platelet count and do not require further intervention; however, more frequently, their platelet counts will again fall and further treatment is required. Splenectomy is still part of the treatment plan for some patients, but more patients receive treatment with an immunosuppressant, such as Rituximab or a TPO receptor agonist. Some patients will receive these treatments sequentially as they lose response to a particular treatment. Clinical guidance published in the UK and the USA suggests that due to the failure or relapse rates after the accepted treatment, further research of novel treatments is required. The mode of action of BMS945429 differs from the available treatment options and as such we are exploring whether it may offer an additional line of therapy in this sometimes difficult to treat patient population.
This trial will assess the safety, effectiveness and the way the drug is absorbed, acts on and is excreted from the body.
This study includes a screening, treatment and follow up period. This is an open label study, which means that both the subject and the study doctor will know the treatment assignment. All subjects will be assigned to BMS 986004. Four (4) doses are being investigated and each dose will be given to a panel of 10 subjects. It is planned to enrol subjects sequentially in an ascending manner to these different doses. Subjects will be given one of the four doses for up to 12 weeks. If sufficient evidence is gathered to judge that one dose is ineffective subjects will be escalated to a higher dose. Subjects are then followed up for 4 weeks at the end of treatment.
Participating patients will undergo: physical examinations, urine and a heart function test, and multiple blood tests.
Globally the study will begin in October 2014 and approximately 40 patients will participate. In the UK the study will begin in January 2015 and is due to end in February 2017. The study is funded by Bristol Myers Squibb.
REC name
North East - Newcastle & North Tyneside 1 Research Ethics Committee
REC reference
14/NE/1175
Date of REC Opinion
24 Nov 2014
REC opinion
Further Information Favourable Opinion