DRKS00017736已完成2 期
Safety and Efficacy of abatacept (s.c.) in patients with CTLA4 insufficiency or LRBA deficiency - ABACHAI
niversitätsklinikum Freiburg vertreten durch den Leitenden Ärztlichen Direktor und die Kaufmännische Direktorin0 个研究点目标入组 20 人开始时间: 2020年7月6日最近更新:
适应症
试验速览
- 阶段
- 2 期
- 状态
- 已完成
- 发起方
- 入组人数
- 20
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
- 分配方式
- N/a: Single Arm Study
- 盲法
- Open (masking not used)
入排标准
- 年龄范围
- 18 Years 至 one（—）
- 性别
- All
入选标准
- •1. Molecular diagnosis of CTLA4 (haplo)-insufficiency or LRBA deficiency with either published mutations or mutations with proven functional effect (impaired CTLA4 staining or CTLA4-dependent transendocytosis).
- •2. Age =18 years.
- •3. IgG serum trough level = 4 g/l (+/- IRT): last test result within 3 months at baseline visit.
- •4. Signed written informed consent.
- •5. Need for intervention on clinical grounds or continued need of therapy with abatacept as evaluated by the treating physician.
- •6. One organ system has to be involved: lung involvement, gut involvement (enteropathy), cytopenias, CNS involvement, lymphoproliferation, immune system, skin involvement. In case of pretreatment with abatacept, organ involvement should be defined using retrospective data from the period before first application of abatacept.
排除标准
- •1. Patient without legal capacity who is unable to understand the nature,
- •significance and consequences of the trial
- •2. Other current immunosuppressive treatments with biologicals or diseasemodifying
- •antirheumatic drugs (DMARDs) other than corticosteroids
- •= 20 mg or abatacept. Between treatment with other biologicals or DMARDs
- •and start of abatacept trial treatment the wash out period of the pretreatment
- •must be kept. In case of pretreatment with rituximab, therapy must be
- •stopped at least 6 month before inclusion to trial.
- •3. Treatment with systemic steroids (prednisolone) in daily dose > 20 mg
- •4. Active or chronic Hepatitis B or tuberculosis infection.
- •5. Active infection or any major episode of infection requiring hospitalization or
- •treatment with intravenous (i.v.) antibiotics within 30 days prior to baseline
- •6. Chronic infection requiring hospitalization or treatment with i.v. antibiotics
- •within 30 days prior to baseline (does not apply for patients already
- •pretreated with abatacept)
- •7. Acute bacterial or viral infection (patients with a chronic and clinically
- •controlled infection can be included).
- •8. Patient on antiviral cytomegalovirus (CMV) prophylaxis within 28 days prior
- •to baseline visit.
- •9. Any malignancies within the last 4 years with the exception of basal cell
- •carcinoma and precancerous conditions (does not apply for patients already
- •pretreated with abatacept)
- •10. Current or planned pregnancy, nursing period
- •11. Epstein-Barr virus (EBV) load of = 5.000 IU/ml or CMV load of = 1.000 IU/ml
- •in plasma at screening
- •12. Receipt of a live virus vaccine within 3 month prior to first application of trial
- •13. Serious uncontrolled concomitant disease not caused by
- •CTLA4 insufficiency or LRBA deficiency.
- •14. Known HIV infection, infectious hepatitis (type A or C) or another
- •uncontrolled infection
- •15. Prior hematopoietic stem cell transplantation (HSCT) or HSCT planned
- •within next 12 months
- •16. Known intolerance to study medication or any of the excipients
研究者
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