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보체억제제 치료 경험이 없거나 최근에 보체억제제 치료를 받지 않은 성인 발작성 야간혈색소뇨증 환자를 대상으로 포젤리맙과 켐디시란 병용요법의 유효성과 안전성을 평가하기 위한 라불리주맙 대조 비열등성 연구 (ACCESS-1)

2026년 11월 4일 업데이트: Regeneron Pharmaceuticals

보체 억제제 치료 경험이 없거나 최근에 보체 억제제 치료를 받지 않은 발작성 야간 혈색소뇨증 환자를 대상으로 포젤리맙과 켐디시란 병용 요법의 효능과 안전성을 평가하기 위한 무작위, 공개, Ravulizumab 대조, 비열등성 연구

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)

연구 개요

상태

완전한

정황

개입 / 치료

연구 유형

중재적

등록 (실제)

202

단계

  • 3단계

연락처 및 위치

이 섹션에서는 연구를 수행하는 사람들의 연락처 정보와 이 연구가 수행되는 장소에 대한 정보를 제공합니다.

연구 장소

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

참여기준

연구원은 적격성 기준이라는 특정 설명에 맞는 사람을 찾습니다. 이러한 기준의 몇 가지 예는 개인의 일반적인 건강 상태 또는 이전 치료입니다.

자격 기준

공부할 수 있는 나이

18년 이상 (성인, 고령자)

건강한 자원 봉사자를 받아들입니다

아니

연구 대상 성별

모두

설명

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

공부 계획

이 섹션에서는 연구 설계 방법과 연구가 측정하는 내용을 포함하여 연구 계획에 대한 세부 정보를 제공합니다.

연구는 어떻게 설계됩니까?

디자인 세부사항

  • 주 목적: 치료
  • 할당: 무작위
  • 중재 모델: 병렬 할당
  • 마스킹: 없음(오픈 라벨)

무기와 개입

참가자 그룹 / 팔
개입 / 치료
실험적: Cohort A

Randomized 1:1
의약품: Ravulizumab

Administered Intravenous (IV) per the protocol

다른 이름들:
  • 울토미리스
  • ALXN1210
의약품: Pozelimab

Administered IV and subcutaneous (SC) per the protocol

다른 이름들:
  • REGN3918
의약품: Cemdisiran

Administered SC per the protocol

다른 이름들:
  • ALN-CC5
실험적: Cohort B

Randomized 1:1
의약품: Pozelimab

Administered IV and subcutaneous (SC) per the protocol

다른 이름들:
  • REGN3918
의약품: Cemdisiran

Administered SC per the protocol

다른 이름들:
  • ALN-CC5
의약품: Eculizumab

Administered IV per the protocol

다른 이름들:
  • 솔리리스

연구는 무엇을 측정합니까?

주요 결과 측정

결과 측정
측정값 설명
기간
Percent change in lactate dehydrogenase (LDH)
기간: From baseline to week 26

Cohort A
From baseline to week 26
Transfusion avoidance
기간: 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
기간: From week 8 through week 26, inclusive

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

2차 결과 측정

결과 측정
측정값 설명
기간
Maintenance of adequate control of hemolysis
기간: From week 8 through week 26, inclusive

Cohort A and B LDH ≤1.5 × ULN
From week 8 through week 26, inclusive
Breakthrough hemolysis
기간: 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
기간: From week 8 through week 26, inclusive

Cohort A LDH ≤1.5 × ULN
From week 8 through week 26, inclusive
Hemoglobin stabilization
기간: 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
기간: 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
기간: 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
기간: 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)
기간: 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
기간: 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
기간: From baseline to week 26

Cohort B
From baseline to week 26
Rate of RBC transfused
기간: 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
기간: 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
기간: Up to Week 26

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

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

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

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

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

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

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

Cohort A and B
From baseline to week 26
Percent change in total CH50
기간: From baseline to week 26

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

Cohort A and B
Up to 60 weeks
Concentrations of total pozelimab in serum
기간: Up to 60 weeks

Cohort A and B
Up to 60 weeks
Concentrations of cemdisiran in plasma
기간: Up to 60 weeks

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

Cohort A
Up to 34 weeks
Concentrations of total eculizumab in serum
기간: Up to 30 weeks

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

Cohort A and B
Up to 60 weeks
Incidence of treatment emergent ADAs to cemdisiran
기간: Up to 60 weeks

Cohort A and B
Up to 60 weeks

공동 작업자 및 조사자

여기에서 이 연구와 관련된 사람과 조직을 찾을 수 있습니다.

스폰서

Regeneron Pharmaceuticals

수사관

  • 연구 책임자: Clinical Trial Management, Regeneron Pharmaceuticals

연구 기록 날짜

이 날짜는 ClinicalTrials.gov에 대한 연구 기록 및 요약 결과 제출의 진행 상황을 추적합니다. 연구 기록 및 보고된 결과는 공개 웹사이트에 게시되기 전에 특정 품질 관리 기준을 충족하는지 확인하기 위해 국립 의학 도서관(NLM)에서 검토합니다.

연구 주요 날짜

연구 시작 (실제)

2022년 8월 1일

기본 완료 (실제)

2026년 9월 28일

연구 완료 (실제)

2026년 10월 29일

연구 등록 날짜

최초 제출

2021년 11월 12일

QC 기준을 충족하는 최초 제출

2021년 11월 12일

처음 게시됨 (실제)

2021년 11월 24일

연구 기록 업데이트

마지막 업데이트 게시됨 (실제)

2026년 11월 6일

QC 기준을 충족하는 마지막 업데이트 제출

2026년 11월 4일

마지막으로 확인됨

2026년 11월 1일

추가 정보

이 연구와 관련된 용어

키워드

추가 관련 MeSH 약관

기타 연구 ID 번호

  • R3918-PNH-2021
  • 2020-004486-40 (EudraCT 번호)
  • 2023 (미국 NIH 보조금/계약)
  • 2023-509657-31-00 (씨티스)

개별 참가자 데이터(IPD) 계획

개별 참가자 데이터(IPD)를 공유할 계획입니까?

예

IPD 계획 설명

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

IPD 공유 기간

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 공유 액세스 기준

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 공유 지원 정보 유형

  • 연구_프로토콜
  • 수액
  • ICF
  • ANALYTIC_CODE
  • CSR

약물 및 장치 정보, 연구 문서

미국 FDA 규제 의약품 연구

예

미국 FDA 규제 기기 제품 연구

아니

이 정보는 변경 없이 clinicaltrials.gov 웹사이트에서 직접 가져온 것입니다. 귀하의 연구 세부 정보를 변경, 제거 또는 업데이트하도록 요청하는 경우 register@clinicaltrials.gov. 문의하십시오. 변경 사항이 clinicaltrials.gov에 구현되는 즉시 저희 웹사이트에도 자동으로 업데이트됩니다. .

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