EUCTR2017-005140-16-BG进行中（未招募）1 期
A Phase IIa, Open Label, Multiple Dose Study to Assess the Safety, Efficacy and Pharmacokinetics of Subcutaneously Administered APL-2 in Subjects with Paroxysmal Nocturnal Hemoglobinuria (PNH)
适应症
试验速览
- 阶段
- 1 期
- 状态
- 进行中（未招募）
- 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 clinical trial of medicinal product
入排标准
- 性别
- All
入选标准
- •1. At least 18 years old (inclusive)
- •2. Diagnosed with PNH (WBC clone >10%)
- •3. Lactose dehydrogenase =2 times the upper limit of normal
- •4. Screening Ferritin = normal and Total Iron Binding Capacity (TIBC) = LLN based on central lab reference ranges. If a subject is receiving iron supplements at screening, the investigator must ensure that his/her dose has been stable for 8 weeks prior to enrolment and must be maintained throughout the study (see Protocol Section 8.4.4)
- •5. Last transfusion within 12 months prior to screening
- •6. Platelet count of >30,000/mm3 at the screening visit
- •7. Absolute neutrophil count >500/ mm3 at the screening visit
- •8. Women of child-bearing potential (WOCBP) must have a negative pregnancy test at screening and must agree to use protocol defined methods of contraception for the duration of the study
- •9. Males must agree to use protocol defined methods of contraception and agree to refrain from donating sperm for the duration of the study
- •10. Vaccination against Neisseria meningitides types A, C, W, Y and B, Streptococcus pneumoniae and Haemophilus influenzae Type B (Hib) either within 2 years prior to Day 1 dosing, or within 14 days after starting treatment with APL-2. Unless documented evidence exists that subjects are non-responders to vaccination as evidenced by titers or display titer levels within acceptable local limits
- •11. Willing and able to give informed consent
- •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 16
- •F.1.3 Elderly (>=65 years) yes
- •F.1.3.1 Number of subjects for this age range 4
排除标准
- •1. Prior eculizumab (Soliris®) treatment
- •2. Active bacterial infection
- •3. Hereditary complement deficiency
- •4. History of bone marrow transplantation
- •5. Concurrent SAA, defined as currently receiving immunosuppressive therapy for SAA including but not limited to cyclosporin A, tacrolimus, mycophenolate mofetil or antithymocyte globulin
- •6. Participation in any other investigational drug trial or exposure to other investigational agent, device or procedure within 30 days
- •7. Evidence of QTcF prolongation defined as >450 ms for males and >470 ms for females at screening
- •8. Breast-feeding women
- •9. History of meningococcal disease
研究者
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