NCT04060264已完成3 期
Randomized, Open-Label, International, Multi-center, Comparative Study of Efficacy and Safety of BCD-148 (JSC BIOCAD, Russia) and Soliris® in Patients With Paroxysmal Nocturnal Hemoglobinuria
适应症
试验速览
- 阶段
- 3 期
- 状态
- 已完成
- 发起方
- Biocad
- 入组人数
- 28
- 试验地点
- 1
- 主要终点
- AUC LDH
研究概览
简要总结
This clinical study is a randomized, open-label, international, multi-center, comparative study of efficacy and safety of BCD-148 and Soliris® in PNH patients.
It is planned to investigate the efficacy, safety, and immunogenicity of one-year eculizumab course in this study.
PNH - Paroxysmal nocturnal hemoglobinuria
研究设计
- 研究类型
- Interventional
- 分配方式
- Randomized
- 干预模型
- Parallel
- 主要目的
- Treatment
- 盲法
- Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
入排标准
- 年龄范围
- 18 Years 至 65 Years（Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •He/she gave written informed consent.
- •Male or female ≥18 and ≤65 years of age.
- •PNH diagnosis documented by flow cytometry data at screening .
- •PNH granulocyte clone size ≥10% (according to flow cytometry performed at screening).
- •Lactate dehydrogenase (LDH) level ≥1.5 times the upper limit of normal (ULN) at screening and at least one of the following symptoms/syndromes: hemoglobinuria, thrombotic complications, transfusion-dependent chronic hemolysis, anemic syndrome, acute kidney injury episodes or chronic kidney disease, pulmonary hypertension, and signs of smooth muscle dystonia (e.g., abdominal pain, dysphagia, erectile dysfunction, and etc.) within three months before informed consent.
- •Platelet count ≥30х109/L at screening.
- •Absolute count of neutrophil granulocytes ≥0.75х109/L at screening.
- •Willingness to undergo vaccination against Neisseria meningitidis during the screening period and at least 14 days before the first administration of an investigational product .
- •If immunosuppressive drug products are used, the duration of this therapy should be at least three months by informed consent date.
- •The willingness of patients and their sexual partners of childbearing potential to use reliable contraception methods starting from the informed consent, throughout the study, and for four weeks after the last dose of an investigational product. This requirement does not apply to patients who underwent surgical sterilization and women with menopause established more than two years ago. Reliable contraception methods include one barrier method in combination with one of the following: spermicides or an intrauterine device.
- •The patient is able, in the Investigator's opinion, to follow study procedures.
排除标准
- •History of meningococcal infection (either well-documented or according to oral information provided by a patient).
- •Other well-documented complement deficiencies (except for those concerning complement component 5).
- •History of bone marrow transplantation (either well-documented or according to oral information provided by a patient).
- •HIV, hepatitis B, active hepatitis C, and syphilis .
- •A patient with newly diagnosed or relapsing aplastic anemia and/or progressive bone marrow failure with indications for allogeneic bone marrow transplantation or combined immunosuppressive therapy within 6 months after informed consent.
- •Acute infection (either well-documented and/or according to oral information provided by a patient) within 4 weeks before informed consent and/or during the screening period and/or relapse of chronic disease at the moment of informed consent and/or during the screening period .
- •Any other chronic diseases present at the time of the informed consent which can negatively affect the patient's safety during the study, in the Investigator' opinion.
- •Use of eculizumab and/other anti-C5 monoclonal antibodies within three months before informed consent .
- •Hypersensitivity to any of BCD-148/Soliris® ingredients, murine proteins and other ingredients of these drug products, and to any of meningococcal vaccine ingredients.
- •Documented malignancy, except for cured basal cell carcinoma or cervical carcinoma in situ .
- •A known alcoholic or drug abuse or signs of present alcoholic/drug abuse that, in the Investigator's opinion, can be a contraindication to treatment with an investigational product or limit treatment compliance.
- •Participation in other clinical studies within 30 days before informed consent and during this study.
- •Pregnancy or lactation or planning for pregnancy/paternity during the clinical study.
结局指标
主要结局
AUC LDH
时间窗: Weeks 5-27
AUC - Area Under Curve of Lactate dehydrogenase
次要结局
- The proportion of patients with stable Hb level during the maintenance therapy period(Weeks 5-27)
- Changes in LDH level over time(week 27, week 52)
- Frequency of breakthrough hemolysis episodes(week 27, week 52)
- Mean Hb level over the maintenance therapy period(Weeks 5-27)
- Change in mean EORTC QLQ-C30 score(week 27, week 52)
- The proportion of patients with AE/SAE related to an investigational product, in the Investigator's opinion(week 27, week 52)
- The proportion of patients with thrombotic complications(week 27, week 52)
- Change in the count of circulating red blood cells with the PNH phenotype RBC(week 27, week 52)
- The proportion of patients who required red blood cell transfusion(week 27, week 52)
- The proportion of patients who discontinued early due to AE/SAE related to an investigational product, in the Investigator's opinion, by arm(week 27, week 52)
- Change in mean FACIT-Fatigue score(week 27, week 52)
- The proportion of patients with СТСАЕ v.5.0 Grade 3-4 AE related to an investigational product, in the Investigator's opinion, by arm(week 27, week 52)
- The proportion of BAb- and NAb-positive patients.(week 27, week 52)
研究者
研究点 (1)
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