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Ravulizumab 对照非劣效性研究评估 Pozelimab 和 Cemdisiran 联合治疗未接受过补体抑制剂治疗或近期未接受过补体抑制剂治疗的阵发性睡眠性血红蛋白尿成年患者的疗效和安全性 (ACCESS-1)

2026年11月4日 更新者：Regeneron Pharmaceuticals

一项随机、开放标签、Ravulizumab 对照、非劣效性研究，以评估 Pozelimab 和 Cemdisiran 联合治疗未接受补体抑制剂治疗或近期未接受补体抑制剂治疗的阵发性睡眠性血红蛋白尿症患者的疗效和安全性

This study is researching a clinical treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH and how the combination compares with 2 existing treatments: ravulizumab and eculizumab.

The pozelimab + cemdisiran combination may be referred to as "study drugs". Ravulizumab and eculizumab may also be called the "comparator drug".

The study is looking at several research questions, including:

  • How effective is the pozelimab + cemdisiran combination compared to ravulizumab?
  • How effective is pozelimab + cemdisiran combination compared to eculizumab?
  • What side effects may happen from taking the study drugs?
  • How much study drugs are in the blood at different times?
  • Whether the body makes antibodies against the study drugs (which could make the study drugs less effective or could lead to side effects)

研究概览

地位

完全的

条件

干预/治疗

研究类型

介入性

注册 (实际的)

202

阶段

  • 第三阶段

联系人和位置

本节提供了进行研究的人员的详细联系信息，以及有关进行该研究的地点的信息。

学习地点

  • 中国
    • Beijing Municipality
      • Beijing、Beijing Municipality、中国、100032
        • Peking Union Medical College Hospital
  • 加拿大
    • Ontario
      • Toronto、Ontario、加拿大、M5G 2C4
        • Toronto General Hospital
  • 匈牙利
      • Budapest、匈牙利、1083
        • Semmelweis University
  • 印度
      • Jaipur、印度、302017
        • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
    • Kerala
      • Kannur、Kerala、印度、670103
        • Malabar Cancer Center, Kerala
      • Kochi、Kerala、印度、682041
        • Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
    • Maharashtra
      • Mumbai、Maharashtra、印度、400022
        • K J Somaiya Super Specialty Hospital & Research Centre
    • National Capital Territory of Delhi
      • New Delhi、National Capital Territory of Delhi、印度、110085
        • Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
    • Punjab
      • Chandigarh、Punjab、印度、708248
        • Postgraduate Institute of Medical Education & Research (PGIMER)
    • Uttar Pradesh
      • Lucknow、Uttar Pradesh、印度、226014
        • Sanjay Gandhi Postgraduate Institute of Medical Sciences
      • Lucknow、Uttar Pradesh、印度、226003
        • King George Hospital
  • 台湾
      • Changhua、台湾、500-06
        • Changhua Christian Hospital
      • Hualien City、台湾、97002
        • Hualien Tzu Chi Hospital
      • Kaohsiung City、台湾、80756
        • Kaohsiung Medical University Hospital
      • Taichung、台湾、4070
        • Taichung Veterans General Hospital (VGHTC)
      • Tainan、台湾、704
        • National Cheng Kung University Hospital
      • Taipei、台湾、114
        • Tri-Service General Hospital
      • Taipei、台湾、100229
        • National Taiwan University Hospital
    • Central Taiwan
      • Taichung、Central Taiwan、台湾、40447
        • China Medical University Hospital
    • Hunan Province
      • Taoyuan、Hunan Province、台湾、33305
        • Chang Gung Memorial Hospital - Linkou Branch
  • 哥伦比亚
    • Antioquia
      • Medellín、Antioquia、哥伦比亚、050034
        • Hospital Pablo Tobon Uribe
  • 土耳其（türkiye）
      • Istanbul、土耳其（türkiye）、34418
        • Istanbul University
      • Izmir、土耳其（türkiye）、35100
        • Ege University
  • 墨西哥
    • Nuevo León
      • Monterrey、Nuevo León、墨西哥、64460
        • Servicio de Hematologia del Hospital Universitario de la Uanl
  • 巴西
      • São Paulo、巴西、08270-070
        • Casa de Saude Santa Marcelina
      • São Paulo、巴西、01321-001
        • A Beneficencia Portuguesa de Sao Paulo, BP Mirante
    • São Paulo
      • Santo André、São Paulo、巴西、09060-650
        • Centro de Estudos e Pesquisas em Hematologia e Oncologia
  • 希腊
      • Thessaloniki、希腊、57010
        • George Papanikolaou Hospital
  • 意大利
      • Turin、意大利、10126
        • Hematology Citta della Salute e della Scienza di Torino
    • Firenze
      • Florence、Firenze、意大利、50139
        • Aou Careggi
    • Roma
      • Rome、Roma、意大利、00168
        • Fondazione Policlinico Universitario A. Gemelli - IRCCS
  • 新加坡
      • Singapore、新加坡、119074
        • National University Hospital
  • 日本
    • Aichi-ken
      • Nagoya、Aichi-ken、日本、466-8650
        • Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
    • Gifu
      • Ōgaki、Gifu、日本、503-8502
        • Ogaki Municipal Hospital
    • Ibaraki
      • Tsukuba、Ibaraki、日本、305-8576
        • University of Tsukuba Hospital
    • Osaka
      • Moriguchi、Osaka、日本、570-8540
        • Matsushita Memorial Hospital
    • Tokyo
      • Shinagawa-ku、Tokyo、日本、141-8625
        • NTT Medical Center Tokyo
  • 波兰
      • Bydgoszcz、波兰、85-168
        • Szpital Uniwersytecki Nr2 Bydgoszcz
      • Warsaw、波兰、02-776
        • Institute of Hematology and Transfusion Medicine
    • Pomeranian Voivodeship
      • Gdansk、Pomeranian Voivodeship、波兰、80-214
        • University Clinical Center Medical University of Gdansk
  • 泰国
      • Bangkok、泰国、10330
        • King Chulalongkorn Memorial Hospital
      • Chiang Mai、泰国、50200
        • Chaing Mai University
      • Khon Kaen、泰国、40002
        • Faculty of Medicine Khon Kaen University
    • Changwat Songkhla
      • Hat Yai、Changwat Songkhla、泰国、90110
        • Clinical Research Center, Faculty of Medicine, Prince of Songkla University
  • 秘鲁
      • Lima、秘鲁、15072
        • Clinica San Felipe
  • 约旦
      • Amman、约旦、11942
        • Jordan University Hospital (JUH)
  • 罗马尼亚
    • Cluj
      • Cluj-Napoca、Cluj、罗马尼亚、400015
        • Ion Chiricuta Oncology Institute
    • Dolj
      • Craiova、Dolj、罗马尼亚、200143
        • Municipal Hospital Filantropia
    • Mureș County
      • Târgu Mureş、Mureș County、罗马尼亚、540136
        • Targu Mures Clinical County Emergency Hospital
  • 美国
    • California
      • Whittier、California、美国、90602
        • The Oncology Institute of Hope & Innovation
  • 英国
      • Leeds、英国、LS9 7TF
        • St James Hospital
  • 菲律宾
    • Central Luzon
      • Quezon、Central Luzon、菲律宾、1011
        • St Lukes Medical Center
  • 西班牙
      • Barcelona、西班牙、08036
        • Hospital Clinic de Barcelona
      • Murcia、西班牙、30008
        • Hospital General JM Morales Meseguer
      • Salamanca、西班牙、37007
        • Hospital Clinico Universitario de Salamanca
    • Vizcaya
      • Bilbao、Vizcaya、西班牙、48013
        • Hospital Universitario Basurto
  • 韩国
      • Busan、韩国、49241
        • Pusan National University Hospital
      • Seoul、韩国、06351
        • Samsung Medical Center
      • Seoul、韩国、03722
        • Severance Hospital
      • Seoul、韩国、07985
        • Ewha Womans University Mokdong Hospital
      • Seoul、韩国、02841
        • Korea University Hospital
      • Seoul、韩国、065791
        • Seoul St. Mary's Hospital - The Catholic University of Korea
    • Gyeonggi-do
      • Suwon、Gyeonggi-do、韩国、16247
        • St. Vincent Hospital
      • Suwon、Gyeonggi-do、韩国、16499
        • Ajou University Medical Center
    • Namdong-Gu
      • Incheon、Namdong-Gu、韩国、21565
        • Gachon University Gil Medical Center
  • 马来西亚
    • Pahang
      • Kuantan、Pahang、马来西亚、25200
        • Hospital Tg Ampuan Afzan
    • Sabah
      • Kota Kinabalu、Sabah、马来西亚、88586
        • Hospital Queen Elizabeth
    • Selangor
      • Ampang、Selangor、马来西亚、68000
        • Hospital Ampang

参与标准

研究人员寻找符合特定描述的人，称为资格标准。这些标准的一些例子是一个人的一般健康状况或先前的治疗。

资格标准

适合学习的年龄

18年 及以上 (成人、年长者)

接受健康志愿者

不

有资格学习的性别

全部

描述

Key Inclusion Criteria:

  1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol
  2. Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol
  3. LDH level ≥2 × ULN at the screening visit
  4. Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol

Key Exclusion Criteria:

  1. Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening
  2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
  3. Body weight <40 kilograms at screening visit
  4. Planned use of any complement inhibitor therapy other than study drugs during the treatment period
  5. Not meeting meningococcal vaccination requirements and, at a minimum documentation of quadrivalent meningococcal vaccination within 5 years prior to the screening visit and serotype B vaccine within 3 years prior to the screening visit as described in the protocol.
  6. Any contraindication for receiving Neisseria meningitidis vaccinations (serotypes ACWY and B).
  7. Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab [Cohort A] or eculizumab [Cohort B] prescribing information, where available, or national guidelines/local practice, or if necessary when administration of the first dose of the quadrivalent meningococcal vaccine [serotype ACWY] or the second dose of the serotype B meningococcal vaccine [when available] is less than 2 weeks prior to study treatment initiation) as described in the protocol
  8. Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
  9. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

学习计划

本节提供研究计划的详细信息，包括研究的设计方式和研究的衡量标准。

研究是如何设计的？

设计细节

  • 主要用途：治疗
  • 分配：随机化
  • 介入模型：并行分配
  • 屏蔽：无（打开标签）

武器和干预

参与者组/臂
干预/治疗
实验性的：Cohort A

Randomized 1:1
药品：Ravulizumab

Administered Intravenous (IV) per the protocol

其他名称：
  • 乌尔托米里斯
  • ALXN1210
药品：Pozelimab

Administered IV and subcutaneous (SC) per the protocol

其他名称：
  • REGN3918
药品：Cemdisiran

Administered SC per the protocol

其他名称：
  • ALN-CC5
实验性的：Cohort B

Randomized 1:1
药品：Pozelimab

Administered IV and subcutaneous (SC) per the protocol

其他名称：
  • REGN3918
药品：Cemdisiran

Administered SC per the protocol

其他名称：
  • ALN-CC5
药品：Eculizumab

Administered IV per the protocol

其他名称：
  • 独角兽

研究衡量的是什么？

主要结果指标

结果测量
措施说明
大体时间
Percent change in lactate dehydrogenase (LDH)
大体时间：From baseline to week 26

Cohort A
From baseline to week 26
Transfusion avoidance
大体时间：From post-baseline day 1 through week 26

Cohort B Not requiring a red blood cell (RBC) transfusion per the protocol
From post-baseline day 1 through week 26
Adequate control of hemolysis
大体时间：From week 8 through week 26, inclusive

Cohort B LDH ≤1.5 × ULN at each visit
From week 8 through week 26, inclusive

次要结果测量

结果测量
措施说明
大体时间
Maintenance of adequate control of hemolysis
大体时间：From week 8 through week 26, inclusive

Cohort A and B LDH ≤1.5 × ULN
From week 8 through week 26, inclusive
Breakthrough hemolysis
大体时间：From post-baseline day 1 through week 26

Cohort A and B LDH ≥2 × ULN per the protocol
From post-baseline day 1 through week 26
Adequate control of hemolysis
大体时间：From week 8 through week 26, inclusive

Cohort A LDH ≤1.5 × ULN
From week 8 through week 26, inclusive
Hemoglobin stabilization
大体时间：From day 1 (post-baseline) through week 26

Cohort A and B Patients who do not receive an RBC transfusion and have no decrease in hemoglobin level per the protocol
From day 1 (post-baseline) through week 26
Normalization of LDH
大体时间：Between week 8 through week 26, inclusive

Cohort A and B LDH ≤1.0 × ULN per the protocol
Between week 8 through week 26, inclusive
Transfusion avoidance
大体时间：Day 1 through week 26

Cohort A and B Not requiring an RBC transfusion as per protocol algorithm based on post-baseline hemoglobin values.
Day 1 through week 26
Change in fatigue as measured by the Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue Scale
大体时间：From baseline to week 26

Cohort A and B FACIT-Fatigue Scale is a 13-item, self-reported PRO measure assessing an individual's level of fatigue during their usual daily activities over the past week. This questionnaire is part of the FACIT measurement system, a compilation of questions measuring health-related quality of life (QoL) in patients with cancer and other chronic illnesses. The FACIT-fatigue assesses the level of fatigue using a Likert scale ranging from 0 (not at all) to 4 (very much). Scores range from 0 to 52, with higher scores indicating greater fatigue.
From baseline to week 26
Change in physical function (PF) scores on the European Organisation for Research and Treatment of Cancer Quality of Life Questionnaire (EORTC-QLQ-C30)
大体时间：From baseline to week 26

Cohort A and B EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
From baseline to week 26
Change in global health status (GHS)/QoL scale score on the EORTC-QLC-C30
大体时间：From baseline to week 26

Cohort A and B EORTC-QLQ-C30 is a 30-item subject self-report questionnaire composed of both multi-item and single scales, including global health status/quality of life, functional Scales (physical, role, emotional, cognitive, and social), symptom scales (fatigue, nausea and vomiting, and pain), and 7 single items (dyspnea, insomnia, appetite loss, constipation, diarrhea, sleep and financial difficulties). Participants rate items on a 4-point scale, with 1 as "not at all" and 4 as "very much."
From baseline to week 26
Percent change in LDH
大体时间：From baseline to week 26

Cohort B
From baseline to week 26
Rate of RBC transfused
大体时间：Post-baseline Day 1 through week 26

Cohort A and B Per protocol algorithm
Post-baseline Day 1 through week 26
Number of units of RBC transfused
大体时间：Post-baseline Day 1 through week 26

Cohort A and B Per protocol algorithm
Post-baseline Day 1 through week 26
Time to first LDH ≤1.5 × ULN
大体时间：Up to Week 26

Cohort A and B
Up to Week 26
Time to first LDH ≤1.0 × ULN
大体时间：Up to Week 26

Cohort A and B
Up to Week 26
Percentage of days with LDH ≤1.5 × ULN
大体时间：Between week 8 and week 26, inclusive

Cohort A and B
Between week 8 and week 26, inclusive
Change in hemoglobin levels
大体时间：From baseline to week 26

Cohort A and B
From baseline to week 26
Incidence and severity of treatment emergent serious adverse events (SAEs)
大体时间：Up to 26 weeks

Cohort A and B
Up to 26 weeks
Incidence and severity of treatment-emergent adverse events (TEAEs) of special interest
大体时间：Up to 26 weeks

Cohort A and B
Up to 26 weeks
Incidence and severity of TEAEs leading to treatment discontinuation
大体时间：Up to 26 weeks

Cohort A and B
Up to 26 weeks
Change in total CH50
大体时间：From baseline to week 26

Cohort A and B
From baseline to week 26
Percent change in total CH50
大体时间：From baseline to week 26

Cohort A and B
From baseline to week 26
Concentration of total C5 in plasma
大体时间：Up to 60 weeks

Cohort A and B
Up to 60 weeks
Concentrations of total pozelimab in serum
大体时间：Up to 60 weeks

Cohort A and B
Up to 60 weeks
Concentrations of cemdisiran in plasma
大体时间：Up to 60 weeks

Cohort A and B
Up to 60 weeks
Concentrations of total ravulizumab in serum
大体时间：Up to 34 weeks

Cohort A
Up to 34 weeks
Concentrations of total eculizumab in serum
大体时间：Up to 30 weeks

Cohort B
Up to 30 weeks
Incidence of treatment emergent anti-drug antibodies (ADAs) to pozelimab
大体时间：Up to 60 weeks

Cohort A and B
Up to 60 weeks
Incidence of treatment emergent ADAs to cemdisiran
大体时间：Up to 60 weeks

Cohort A and B
Up to 60 weeks

合作者和调查者

在这里您可以找到参与这项研究的人员和组织。

赞助

Regeneron Pharmaceuticals

调查人员

  • 研究主任：Clinical Trial Management、Regeneron Pharmaceuticals

研究记录日期

这些日期跟踪向 ClinicalTrials.gov 提交研究记录和摘要结果的进度。研究记录和报告的结果由国家医学图书馆 (NLM) 审查，以确保它们在发布到公共网站之前符合特定的质量控制标准。

研究主要日期

学习开始 (实际的)

2022年8月1日

初级完成 (实际的)

2026年9月28日

研究完成 (实际的)

2026年10月29日

研究注册日期

首次提交

2021年11月12日

首先提交符合 QC 标准的

2021年11月12日

首次发布 (实际的)

2021年11月24日

研究记录更新

最后更新发布 (实际的)

2026年11月6日

上次提交的符合 QC 标准的更新

2026年11月4日

最后验证

2026年11月1日

更多信息

与本研究相关的术语

关键字

其他相关的 MeSH 术语

其他研究编号

  • R3918-PNH-2021
  • 2020-004486-40 (EudraCT编号)
  • 2023 (美国 NIH 拨款/合同)
  • 2023-509657-31-00 (克蒂斯)

计划个人参与者数据 (IPD)

计划共享个人参与者数据 (IPD)？

是的

IPD 计划说明

All Individual Patient Data (IPD) that underlie publicly available results will be considered for sharing

IPD 共享时间框架

When Regeneron has:

  • received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development
  • made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry),
  • the legal authority to share the data, and
  • ensured the ability to protect participant privacy.

IPD 共享访问标准

Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

IPD 共享支持信息类型

  • 研究方案
  • 树液
  • 国际碳纤维联合会
  • 分析代码
  • 企业社会责任

药物和器械信息、研究文件

研究美国 FDA 监管的药品

是的

研究美国 FDA 监管的设备产品

不

此信息直接从 clinicaltrials.gov 网站检索，没有任何更改。如果您有任何更改、删除或更新研究详细信息的请求，请联系 register@clinicaltrials.gov. clinicaltrials.gov 上实施更改，我们的网站上也会自动更新.

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