A Study to Investigate Belimumab for the Treatment of Chronic Immune Thrombocytopenia.
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.
Purpura, Thrombocytopaenic, Idiopathic
Drug: Normal saline placebo
|Study Design:||Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Subject, Caregiver, Investigator)
|Official Title:||A Clinical and Mechanistic Proof of Efficacy Study With Belimumab in Chronic Immune Thrombocytopenia (ITP) Patients.|
- Platelet count [ Time Frame: Baseline, week 28 ]Change in platelet count
- Anti-platelet autoantibodies [ Time Frame: Baseline, week 28 ]Change in anti-platelet autoantibodies
- Platelet count (time) [ Time Frame: Baseline, week 0, 2, 4, 8, 12, 16, 20, 24, 28 ]Time to first response of platelet count increase >20,000/microlitre (μl) from baseline
- Platelet count (incidence) [ Time Frame: Baseline, week 28 ]Incidence of response of platelet count increase >20,000/μL from baseline
- Platelet count (incidence of complete response) [ Time Frame: Baseline, week 28 ]Incidence of complete response as defined by platelet count to >100,000
- Platelet count (incidence of doubling) [ Time Frame: Baseline, week 28 ]Incidence of subjects with ≥2 times baseline platelet count
- Vital signs [ Time Frame: Baseline, week 0, 2, 4, 8, 12, 16, 20, 24, 28, 40 ]Change in vital signs outside normal range
- Clinical chemistry and haematology [ Time Frame: Baseline, week 2, 4, 8, 12, 16, 20, 24, 28, 40 ]Change in clinical chemistry and haematology
- Immunogenicity [ Time Frame: Baseline, week 12, 28, 40, 52 ]Change in immunogenicity
- Serum concentrations of belimumab [ Time Frame: Baseline, week 2, 8, 24, 28, 40, 52 ]Change in serum concentrations of belimumab
- Serum and/or platelet bound anti-platelet antibodies [ Time Frame: Baseline, week 4, 8, 12, 16, 20, 24, 28, 40, 52 ]Change in serum and/or platelet bound anti-platelet antibodies
- B cell and T cell sub-populations and B lymphocyte stimulator (BLyS) receptor [ Time Frame: Baseline, week 4, 8, 16, 24, 40, 52 ]Change in B cell and T cell sub-populations and BLyS receptor
- Antigen-specific B cells and T cells [ Time Frame: Baseline, week 8, 16, 24, 40 ]Change in antigen-specific B cells and T cells
- Serum cytokine/chemokine profile [ Time Frame: Baseline, week 8, 16, 24, 28, 40 ]Change in serum cytokine/chemokine profile
- Transcriptome profile [ Time Frame: Baseline, week 8, 28 ]Change in transcriptome profile
- Autoantibody profile [ Time Frame: Baseline, week 28 ]Change in autoantibody profile
|Study Start Date:||March 2013|
|Estimated Study Completion Date:||April 2015|
|Estimated Primary Completion Date:||April 2015 (Final data collection date for primary outcome measure)|
Reconstituted solution for intravenous infusion
10 mg /kg given as an intravenous infusion every 4 weeks for 24 weeks (with an additional dose at Week 2)
Other Name: Benlysta/LymphoStat-B
Placebo Comparator: Normal saline
Solution for intravenous infusion
Drug: Normal saline placebo
Placebo given as an intravenous infusion every 4 weeks for 24 weeks (with an additional dose at Week 2)
Other Name: Saline
Please refer to this study by its ClinicalTrials.gov identifier: NCT01440361
|Study Director:||GSK Clinical Trials||GlaxoSmithKline|