PER-014-19招募中未知
A PHASE 3, RANDOMIZED, MULTICENTER, OPEN-LABEL, CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF APL-2 IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA (PNH)
Apellis Pharmaceuticals Inc.,0 个研究点目标入组 9 人开始时间: 2019年7月10日最近更新:
试验速览
- 阶段
- 未知
- 状态
- 招募中
- 发起方
- 入组人数
- 9
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
入选标准
- •At Visit 1 screening, subjects must fulfill all of the following inclusion criteria to be eligible for participation in the study:
- •1.Be at least 18 years old (inclusive).
- •2.Have LDH ≥1.5 x ULN at the screening visit.
- •3.Have PNH diagnosis, confirmed by high sensitivity flow cytometry (granulocyte or monocyte clone >10%).
- •4.Have Hb less than the lower limit of normal (LLN) at the screening visit.
- •5.Have ferritin greater than/equal to the LLN, or total iron binding capacity (TIBC) less than/equal to ULN at the screening visit, based on central laboratory reference ranges. If a subject is receiving iron supplements at screening, the Investigator must ensure that the subject’s dose has been stable for 4 weeks prior to screening, and it must be maintained throughout the study. Subjects not receiving iron at screening must not start iron supplementation during the course of the study.
- •6.Body mass index (BMI) ≤ 35 kg/m2 at the screening visit.
- •7.Have a platelet count of >50,000/mm3 at the screening visit.
- •8.Have an absolute neutrophil count >500/mm3 at the screening visit.
- •9.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 and for 90 days after their last dose of study drug.
- •10.Males must agree to use protocol-defined methods of contraception and agree to refrain from donating sperm for the duration of the study and for 90 days after their last dose of study drug.
- •11.Have vaccination against Streptococcus pneumoniae, Neisseria meningitides (types A, C, W, Y, and B), and Haemophilus influenzae (type B) either within 2 years prior to Day 1 dosing, or agree to receive vaccination 14 days after starting treatment with APL-2 (along with prophylactic antibiotic therapy for at least the 14 days between APL-2 treatment initiation and vaccination and for 14 days post vaccination). Vaccination is mandatory, unless documented evidence exists that subjects are non-responders to vaccination (as evidenced by titers or display titer levels within acceptable local limits).
- •12.Be willing and able to give informed consent.
排除标准
- •Subjects will be excluded from the study if there is evidence of any of the following criteria at screening:
- •1.Treatment with any complement inhibitor (eg, eculizumab) within 3 months prior to screening.
- •2.Hereditary complement deficiency.
- •3.History of bone marrow transplantation.
- •4.Concomitant use of any of the following medications is prohibited if not on a stable regimen for the time period indicated below prior to screening:
- •Erythropoietin or immunosuppressants for at least 8 weeks
- •Systemic corticosteroids for at least 4 weeks
- •Vitamin K antagonists (eg, warfarin) with a stable international normalized ratio (INR) for at least 4 weeks
- •Iron supplements, vitamin B12, or folic acid for at least 4 weeks
- •Low-molecular-weight heparin for at least 4 weeks
- •5.History or presence of hypersensitivity or idiosyncratic reaction to compounds related to the investigational product or SC administration.
- •6.Participation in any other investigational drug trial or exposure to other investigational agent, device, or procedure within 30 days or 5 half-lives, whichever is longer.
- •7.Planning to become pregnant or currently a breast-feeding woman.
- •8.History of meningococcal disease.
- •9.Any comorbidity or condition (such as malignancy) that, in the opinion of the Investigator, could put the subject at increased risk or potentially confound study data.
研究者
相似试验
进行中（未招募）
1 期
A Phase 3, Open-Label, Study to Evaluate Pharmacokinetics and Pharmacodynamics of Edoxaban and to compare the safety and efficacy of Edoxaban with standard of care treatment in Paediatric Patients confirmed as requiring treatment for a blood clotMedDRA version: 21.1Level: LLTClassification code 10066899Term: Venous thromboembolismSystem Organ Class: 100000004866venous thromboembolismEUCTR2016-000991-49-PTDaiichi Sankyo, Inc.290
进行中（未招募）
1 期
A Phase 3, Open-Label, Study to Evaluate Pharmacokinetics and Pharmacodynamics of Edoxaban and to compare the safety and efficacy of Edoxaban with standard of care treatment in Paediatric Patients confirmed as requiring treatment for a blood clotMedDRA version: 19.0Level: LLTClassification code 10066899Term: Venous thromboembolismSystem Organ Class: 100000004866venous thromboembolismEUCTR2016-000991-49-ESDaiichi Sankyo, Inc.274
进行中（未招募）
1 期
A Phase 3, Open-Label, Study to Evaluate Pharmacokinetics and Pharmacodynamics of Edoxaban and to compare the safety and efficacy of Edoxaban with standard of care treatment in Paediatric Patients confirmed as requiring treatment for a blood clotvenous thromboembolismEUCTR2016-000991-49-DEDaiichi Sankyo, Inc.274
进行中（未招募）
1 期
A Phase 3, Open-Label, Study to Evaluate Pharmacokinetics and Pharmacodynamics of Edoxaban and to compare the safety and efficacy of Edoxaban with standard of care treatment in Paediatric Patients confirmed as requiring treatment for a blood clotMedDRA version: 21.1Level: LLTClassification code 10066899Term: Venous thromboembolismSystem Organ Class: 100000004866venous thromboembolismEUCTR2016-000991-49-NLDaiichi Sankyo, Inc.274
进行中（未招募）
1 期
A Phase 3, Open-Label, Study to Evaluate Pharmacokinetics and Pharmacodynamics of Edoxaban and to compare the safety and efficacy of Edoxaban with standard of care treatment in Paediatric Patients confirmed as requiring treatment for a blood clotMedDRA version: 21.1Level: LLTClassification code 10066899Term: Venous thromboembolismSystem Organ Class: 100000004866venous thromboembolismEUCTR2016-000991-49-PLDaiichi Sankyo, Inc.290