A Randomized, Double-blind Superiority Trial to Evaluate the Safety, Tolerability, and Efficacy of Belimumab in Refractory ITP Subjects
- Conditions
- Autoimmune thrombocytopenia (ITP) is a common disorder in which platelets are destroyed by autoantibodies. BAFF has been shown to be elevated in the serum of ITP patients. Blockage of BAFF has been shown to be efficient in other autoimmune diseases. Here, we want to investigate the efficiency and tolerability of Belimumab in the treatment of patients with ITP.
- Registration Number
- EUCTR2007-003642-15-DE
- Lead Sponsor
- Charité Universitätsmedizin Berlin
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- Not specified
•Age 18 – 70 years
•Gender: male or female
•Diagnosed ITP for at least 6 months prior to screening, and have a platelet count of <30.000 platelets/mikroliter on Day 1.
•Refractory patients who have either not responded to one or more prior therapies, or have relapsed within 3 months prior therapy. Previous therapies include corticosteroids, IVIG, anti-D immunoglobulin, azathioprine, danazol, cyclophosphamide, immunomodulators and/or splenectomy. Previous therapies with immunoglobulins, immunomodulators and cyclophosphamide must have been completed at least two weeks prior to randomization.
•Subjects with maintenance immunosuppressive therapy (corticosteroids, azathioprine, danazol, cyclosporine A, MMF) and inadequate increase of platelets (<30,000/mikroliter).
•Subjects treated with maintenance immunosuppressive therapy (corticosteroids, danazol, cyclosporine A) must be receiving a dose that has been stable for at least 1 month.
•Normal protrombin time (PTT) and activated partial thromboplastin time (aPTT).
•A complete blood count, reticulocyte count, creatinine, ALT, AST, bilirubin within the reference range with the following exceptions:
•Platelet count < 30.000/mikroliter
•Hemoglobin: female and male >= 10.0 g/dL are eligible for inclusion.
•ANC>=1500/mikroliter is required for inclusion; elevated WBC/ANC due to steroid treatment is acceptable.
•Subject is practicing an acceptable method of contraception (documented in chart). Female subject or female partners of male subjects must either be on non-childbearing potential (hysterectomy, bilateral, oophrenectomy, bilateral tube ligation or post-menopausal >=1 year).
•Complete abstinence of intercourse
•Intrauterine device
•Two forms of barriers contraception
•Male partner is sterile and the one partner of the female. Contraception: if subject is male – partner is sterile, if female – subject sterile.
•Systemic contraceptives (combined or progesterone only)
•Subject is able to understand and comply with the protocol requirements and instructions and intends to complete the study as planned
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range
•History of thrombosis within the last year.
•Pre-existing cardiac disease; myocardial infarction in the last three months
•Female subjects who are nursing or pregnant (positive serum or urine
•Subject has consumed aspirin, aspirin containing compounds, anti-coagulants or non-steroidal anti-inflammatories for > 3 consecutive days within 2 weeks during the study.
•Any laboratory or clinical evidence for HIV, Hepatitis C, chronic Hepatitis B infection beta-chorionic gonatrotropin pregnancy test) at screening at pre-dose on Day 1.
•History of alcohol/drug abuse or dependence within 12 months of the study.
•Treatment with an investigational drug within 30 days or 5 half-lives preceding the first dose of study medication.
•A complete blood count (CBC) and/or reticulocyte count outside the reference range , with the following exceptions:
•Platelet count < 30.000/mikroliter is required for inclusion.
•Haemoglobin: females and males >=10 g/dL are eligible for inclusion.
•ANC>=1500/mikroliter (1.5 x 109/L) is eligible for inclusion.
•History of platelet aggregation that prevents reliable measuring of platelet counts.
•An laboratory or clinical evidence for HIV infection; an clinical history or laboratory evidence for hepatitis C infection; an clinical history or laboratory evidence for hepatitis B infection; or any evidence for active hepatitis at the time of subject screening; if the potential subject has no clinical history that would support HIV infection or hepatitis infection, no further laboratory screening is necessary; however standard medical practice would suggest evaluation of patients who have risk factors for these infections.
•Persons who are detained officially or legally to an official institute
•Women of childbearing age without highly effective contraception (PEARL-Index < 1%)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method Main Objective: To evaluate the efficacy of belimumab in refractory ITP subjects when administered every 4 weeks to adult patients with chronic ITP. <br><br>;Secondary Objective: To assess the impact of Belimumab on the incidence and severity of symptoms of thrombocytopenia compared to standard treatment when administered every 4 weeks to adult patients with chronic ITP. <br><br>To assess the impact of Belimumab on the equality of life when administered every 4 weeks to adult patients with chronic ITP. <br><br>To assess the impact of Belimumab on the plasma levels of soluble BAFF in adult patients with chronic ITP. <br><br>;Primary end point(s): - Platelet count after 56 days of therapy with belimumab
- Secondary Outcome Measures
Name Time Method