Chronic immune thrombocytopenia (ITP) is a longterm disease in which the blood does not clot normally. This is due to a low number of blood cell fragments called platelets. Platelets clot to seal small cuts or breaks on blood vessel walls and stop bleeding. Normally the immune system makes proteins called antibodies to fight off harmful substances that enter the body. In ITP, the immune system produces antibodies that attack and destroy the body's platelets by mistake. Patients can suffer from bleeding under the skin, nosebleeds, blood in urine or stools and in very severe cases bleeding in the brain. Patients have an increased frequency of death from bleeding complications compared to normal. Chronic ITP is fairly rare , with an incidence of 32 new cases/million people each year. Existing treatments work by lowering the activity of the immune system or directly increasing platelet count. These treatments do not work effectively in all patients and can have side effects. We hope that understanding how belimumab works in ITP will help in the development of future treatments for ITP and other autoimmune diseases. We will test the safety, blood levels and effects of the study medication in people with chronic ITP. Patients will receive the study medication intravenously (through a needle inserted into a vein) and blood samples will be taken before and on several occasions afterwards. Up to 40 patients with chronic ITP, aged 18 to 75 will participate. Approximately 11 patients will take dummy medicine instead of the study medicine neither they or their study doctor will know which one they are given. Participants will take up to 57 weeks to finish the study. They'll make 12 outpatient visits. The study will take place in hospitals in the UK. Other sites in mainland Europe may also be initiated. A pharmaceutical company, GlaxoSmithKline, is funding the study.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Masking
TRIPLE
10 mg /kg given as an intravenous infusion every 4 weeks for 24 weeks (with an additional dose at Week 2)
Placebo given as an intravenous infusion every 4 weeks for 24 weeks (with an additional dose at Week 2)
Platelet count
Change in platelet count
Time frame: Baseline, week 28
Anti-platelet autoantibodies
Change in anti-platelet autoantibodies
Time frame: Baseline, week 28
Platelet count (time)
Time to first response of platelet count increase \>20,000/microlitre (μl) from baseline
Time frame: Baseline, week 0, 2, 4, 8, 12, 16, 20, 24, 28
Platelet count (incidence)
Incidence of response of platelet count increase \>20,000/μL from baseline
Time frame: Baseline, week 28
Platelet count (incidence of complete response)
Incidence of complete response as defined by platelet count to \>100,000
Time frame: Baseline, week 28
Platelet count (incidence of doubling)
Incidence of subjects with ≥2 times baseline platelet count
Time frame: Baseline, week 28
Vital signs
Change in vital signs outside normal range
Time frame: Baseline, week 0, 2, 4, 8, 12, 16, 20, 24, 28, 40
Clinical chemistry and haematology
Change in clinical chemistry and haematology
Time frame: Baseline, week 2, 4, 8, 12, 16, 20, 24, 28, 40
Immunogenicity
Change in immunogenicity
Time frame: Baseline, week 12, 28, 40, 52
Serum concentrations of belimumab
Change in serum concentrations of belimumab
Time frame: Baseline, week 2, 8, 24, 28, 40, 52
Serum and/or platelet bound anti-platelet antibodies
Change in serum and/or platelet bound anti-platelet antibodies
Time frame: Baseline, week 4, 8, 12, 16, 20, 24, 28, 40, 52
B cell and T cell sub-populations and B lymphocyte stimulator (BLyS) receptor
Change in B cell and T cell sub-populations and BLyS receptor
Time frame: Baseline, week 4, 8, 16, 24, 40, 52
Antigen-specific B cells and T cells
Change in antigen-specific B cells and T cells
Time frame: Baseline, week 8, 16, 24, 40
Serum cytokine/chemokine profile
Change in serum cytokine/chemokine profile
Time frame: Baseline, week 8, 16, 24, 28, 40
Transcriptome profile
Change in transcriptome profile
Time frame: Baseline, week 8, 28
Autoantibody profile
Change in autoantibody profile
Time frame: Baseline, week 28
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.