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補体阻害剤治療未経験または最近補体阻害剤治療を受けていない発作性夜間ヘモグロビン尿症の成人患者におけるポゼリマブとセムジシラン併用療法の有効性と安全性を評価するためのラブリズマブ対照非劣性研究 (ACCESS-1)

2026年11月4日 更新者：Regeneron Pharmaceuticals

補体阻害剤治療を受けていない、または最近補体阻害剤治療を受けていない発作性夜間ヘモグロビン尿症患者におけるポゼリマブとセムジシラン併用療法の有効性と安全性を評価する無作為化非盲検ラブリズマブ対照非劣性研究

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)

調査の概要

状態

完了

条件

介入・治療

研究の種類

介入

入学 (実際)

202

段階

  • フェーズ 3

連絡先と場所

このセクションには、調査を実施する担当者の連絡先の詳細と、この調査が実施されている場所に関する情報が記載されています。

研究場所

  • アメリカ
    • California
      • Whittier、California、アメリカ、90602
        • The Oncology Institute of Hope & Innovation
  • イギリス
      • Leeds、イギリス、LS9 7TF
        • St James Hospital
  • イタリア
      • Turin、イタリア、10126
        • Hematology Citta della Salute e della Scienza di Torino
    • Firenze
      • Florence、Firenze、イタリア、50139
        • Aou Careggi
    • Roma
      • Rome、Roma、イタリア、00168
        • Fondazione Policlinico Universitario A. Gemelli - IRCCS
  • インド
      • Jaipur、インド、302017
        • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
    • Kerala
      • Kannur、Kerala、インド、670103
        • Malabar Cancer Center, Kerala
      • Kochi、Kerala、インド、682041
        • Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
    • Maharashtra
      • Mumbai、Maharashtra、インド、400022
        • K J Somaiya Super Specialty Hospital & Research Centre
    • National Capital Territory of Delhi
      • New Delhi、National Capital Territory of Delhi、インド、110085
        • Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
    • Punjab
      • Chandigarh、Punjab、インド、708248
        • Postgraduate Institute of Medical Education & Research (PGIMER)
    • Uttar Pradesh
      • Lucknow、Uttar Pradesh、インド、226014
        • Sanjay Gandhi Postgraduate Institute of Medical Sciences
      • Lucknow、Uttar Pradesh、インド、226003
        • King George Hospital
  • カナダ
    • Ontario
      • Toronto、Ontario、カナダ、M5G 2C4
        • Toronto General Hospital
  • ギリシャ
      • Thessaloniki、ギリシャ、57010
        • George Papanikolaou Hospital
  • コロンビア
    • Antioquia
      • Medellín、Antioquia、コロンビア、050034
        • Hospital Pablo Tobon Uribe
  • シンガポール
      • Singapore、シンガポール、119074
        • National University Hospital
  • スペイン
      • Barcelona、スペイン、08036
        • Hospital Clinic de Barcelona
      • Murcia、スペイン、30008
        • Hospital General JM Morales Meseguer
      • Salamanca、スペイン、37007
        • Hospital Clinico Universitario de Salamanca
    • Vizcaya
      • Bilbao、Vizcaya、スペイン、48013
        • Hospital Universitario Basurto
  • タイ
      • Bangkok、タイ、10330
        • King Chulalongkorn Memorial Hospital
      • Chiang Mai、タイ、50200
        • Chaing Mai University
      • Khon Kaen、タイ、40002
        • Faculty of Medicine Khon Kaen University
    • Changwat Songkhla
      • Hat Yai、Changwat Songkhla、タイ、90110
        • Clinical Research Center, Faculty of Medicine, Prince of Songkla University
  • トルコ（Türkiye）
      • Istanbul、トルコ（Türkiye）、34418
        • Istanbul University
      • Izmir、トルコ（Türkiye）、35100
        • Ege University
  • ハンガリー
      • Budapest、ハンガリー、1083
        • Semmelweis University
  • フィリピン
    • Central Luzon
      • Quezon、Central Luzon、フィリピン、1011
        • St Lukes Medical Center
  • ブラジル
      • São Paulo、ブラジル、08270-070
        • Casa de Saude Santa Marcelina
      • São Paulo、ブラジル、01321-001
        • A Beneficencia Portuguesa de Sao Paulo, BP Mirante
    • São Paulo
      • Santo André、São Paulo、ブラジル、09060-650
        • Centro de Estudos e Pesquisas em Hematologia e Oncologia
  • ペルー
      • Lima、ペルー、15072
        • Clinica San Felipe
  • ポーランド
      • Bydgoszcz、ポーランド、85-168
        • Szpital Uniwersytecki Nr2 Bydgoszcz
      • Warsaw、ポーランド、02-776
        • Institute of Hematology and Transfusion Medicine
    • Pomeranian Voivodeship
      • Gdansk、Pomeranian Voivodeship、ポーランド、80-214
        • University Clinical Center Medical University of Gdansk
  • マレーシア
    • Pahang
      • Kuantan、Pahang、マレーシア、25200
        • Hospital Tg Ampuan Afzan
    • Sabah
      • Kota Kinabalu、Sabah、マレーシア、88586
        • Hospital Queen Elizabeth
    • Selangor
      • Ampang、Selangor、マレーシア、68000
        • Hospital Ampang
  • メキシコ
    • Nuevo León
      • Monterrey、Nuevo León、メキシコ、64460
        • Servicio de Hematologia del Hospital Universitario de la Uanl
  • ヨルダン
      • Amman、ヨルダン、11942
        • Jordan University Hospital (JUH)
  • ルーマニア
    • Cluj
      • Cluj-Napoca、Cluj、ルーマニア、400015
        • Ion Chiricuta Oncology Institute
    • Dolj
      • Craiova、Dolj、ルーマニア、200143
        • Municipal Hospital Filantropia
    • Mureș County
      • Târgu Mureş、Mureș County、ルーマニア、540136
        • Targu Mures Clinical County Emergency Hospital
  • 中国
    • Beijing Municipality
      • Beijing、Beijing Municipality、中国、100032
        • Peking Union Medical College 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
  • 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
  • 韓国
      • Busan、韓国、49241
        • Pusan National University Hospital
      • Seoul、韓国、06351
        • Samsung Medical Center
      • Seoul、韓国、03722
        • Severance Hospital
      • Seoul、韓国、07985
        • Ewha Womans University Mokdong Hospital
      • Seoul、韓国、02841
        • Korea University Hospital
      • Seoul、韓国、065791
        • Seoul St. Mary's Hospital - The Catholic University of Korea
    • Gyeonggi-do
      • Suwon、Gyeonggi-do、韓国、16247
        • St. Vincent Hospital
      • Suwon、Gyeonggi-do、韓国、16499
        • Ajou University Medical Center
    • Namdong-Gu
      • Incheon、Namdong-Gu、韓国、21565
        • Gachon University Gil Medical Center

参加基準

研究者は、適格基準と呼ばれる特定の説明に適合する人を探します。これらの基準のいくつかの例は、人の一般的な健康状態または以前の治療です。

適格基準

就学可能な年齢

18年歳以上 (大人、高齢者)

健康ボランティアの受け入れ

いいえ

受講資格のある性別

全て

説明

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

研究計画

このセクションでは、研究がどのように設計され、研究が何を測定しているかなど、研究計画の詳細を提供します。

研究はどのように設計されていますか？

デザインの詳細

  • 主な目的：処理
  • 割り当て：ランダム化
  • 介入モデル：並列代入
  • マスキング：なし（オープンラベル）

武器と介入

参加者グループ / アーム
介入・治療
実験的：Cohort A

Randomized 1:1
薬：Ravulizumab

Administered Intravenous (IV) per the protocol

他の名前：
  • ウルトミリス
  • ALXN1210
薬：Pozelimab

Administered IV and subcutaneous (SC) per the protocol

他の名前：
  • REGN3918
薬：Cemdisiran

Administered SC per the protocol

他の名前：
  • アルン-CC5
実験的：Cohort B

Randomized 1:1
薬：Pozelimab

Administered IV and subcutaneous (SC) per the protocol

他の名前：
  • REGN3918
薬：Cemdisiran

Administered SC per the protocol

他の名前：
  • アルン-CC5
薬：Eculizumab

Administered IV per the protocol

他の名前：
  • ソリリス

この研究は何を測定していますか？

主要な結果の測定

結果測定
メジャーの説明
時間枠
Percent change in lactate dehydrogenase (LDH)
時間枠：From baseline to week 26

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

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

二次結果の測定

結果測定
メジャーの説明
時間枠
Maintenance of adequate control of hemolysis
時間枠：From week 8 through week 26, inclusive

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

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

Cohort A and B
Up to 60 weeks

協力者と研究者

ここでは、この調査に関係する人々や組織を見つけることができます。

スポンサー

Regeneron Pharmaceuticals

捜査官

  • スタディディレクター：Clinical Trial Management、Regeneron Pharmaceuticals

研究記録日

これらの日付は、ClinicalTrials.gov への研究記録と要約結果の提出の進捗状況を追跡します。研究記録と報告された結果は、国立医学図書館 (NLM) によって審査され、公開 Web サイトに掲載される前に、特定の品質管理基準を満たしていることが確認されます。

主要日程の研究

研究開始 (実際)

2022年8月1日

一次修了 (実際)

2026年9月28日

研究の完了 (実際)

2026年10月29日

試験登録日

最初に提出

2021年11月12日

QC基準を満たした最初の提出物

2021年11月12日

最初の投稿 (実際)

2021年11月24日

学習記録の更新

投稿された最後の更新 (実際)

2026年11月6日

QC基準を満たした最後の更新が送信されました

2026年11月4日

最終確認日

2026年11月1日

詳しくは

本研究に関する用語

キーワード

追加の関連 MeSH 用語

その他の研究ID番号

  • R3918-PNH-2021
  • 2020-004486-40 (EudraCT番号)
  • 2023 (米国 NIH グラント/契約)
  • 2023-509657-31-00 (Ctis)

個々の参加者データ (IPD) の計画

個々の参加者データ (IPD) を共有する予定はありますか?

はい

IPD プランの説明

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

IPD 共有時間枠

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 共有アクセス基準

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 共有サポート情報タイプ

  • STUDY_PROTOCOL
  • SAP
  • ICF
  • ANALYTIC_CODE
  • CSR

医薬品およびデバイス情報、研究文書

米国FDA規制医薬品の研究

はい

米国FDA規制機器製品の研究

いいえ

この情報は、Web サイト clinicaltrials.gov から変更なしで直接取得したものです。研究の詳細を変更、削除、または更新するリクエストがある場合は、register@clinicaltrials.gov。 までご連絡ください。 clinicaltrials.gov に変更が加えられるとすぐに、ウェブサイトでも自動的に更新されます。

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