A Study to Evaluate How Safe Pozelimab + Cemdisiran Combination Therapy is and How Well it Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Who Have Not Recently Received or Have Not Received Complement Inhibitor Treatment

Last updated: February 20, 2026
Sponsor: Regeneron Pharmaceuticals
Overall Status: Active - Recruiting

Phase

3

Condition

White Cell Disorders

Hemoglobinuria, Paroxysmal

Aplastic Anemia

Treatment

Cemdisiran

Pozelimab

Eculizumab

Clinical Study ID

NCT05133531
R3918-PNH-2021
2023-509657-31-00
2020-004486-40
  • Ages > 18
  • All Genders

Study Summary

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)

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

  1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNHgranulocytes or monocytes as described in the protocol

  2. Active disease, as defined by the presence of 1 or more PNH-related signs orsymptoms 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 perprotocol

Exclusion

Key Exclusion Criteria:

  1. Prior treatment with eculizumab within 3 months prior to screening, ravulizumabwithin 6 months prior to screening, or other complement inhibitors within 5half-lives of the respective agent prior to screening

  2. Receipt of an organ transplant, history of bone marrow transplantation or otherhematologic transplant

  3. Body weight <40 kilograms at screening visit

  4. Planned use of any complement inhibitor therapy other than study drugs during thetreatment period

  5. Not meeting meningococcal vaccination requirements and, at a minimum documentationof quadrivalent meningococcal vaccination within 5 years prior to the screeningvisit and serotype B vaccine within 3 years prior to the screening visit asdescribed in the protocol.

  6. Any contraindication for receiving Neisseria meningitidis vaccinations (serotypesACWY and B).

  7. Unable to take antibiotics for meningococcal prophylaxis (if required by localravulizumab [Cohort A] or eculizumab [Cohort B] prescribing information, whereavailable, or national guidelines/local practice, or if necessary whenadministration of the first dose of the quadrivalent meningococcal vaccine [serotypeACWY] or the second dose of the serotype B meningococcal vaccine [when available] isless than 2 weeks prior to study treatment initiation) as described in the protocol

  8. Any active, ongoing infection or a recent infection requiring ongoing systemictreatment with antibiotics, antivirals, or antifungals within 2 weeks of screeningor during the screening period

  9. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Study Design

Total Participants: 190
Treatment Group(s): 4
Primary Treatment: Cemdisiran
Phase: 3
Study Start date:
August 01, 2022
Estimated Completion Date:
January 28, 2027

Connect with a study center

  • Centro de Estudos e Pesquisas em Hematologia e Oncologia

    Santo André 3449701, São Paulo 3448433 09060-650
    Brazil

    Active - Recruiting

  • A Beneficencia Portuguesa de Sao Paulo, BP Mirante

    São Paulo 3448439, 01321-001
    Brazil

    Active - Recruiting

  • Casa de Saude Santa Marcelina

    São Paulo 3448439, 08270-070
    Brazil

    Active - Recruiting

  • Toronto General Hospital

    Toronto, Ontario M5G2 C4
    Canada

    Site Not Available

  • Toronto General Hospital

    Toronto 6167865, Ontario 6093943 M5G 2C4
    Canada

    Active - Recruiting

  • Peking Union Medical College Hospital

    Beijing, Beijing 100032
    China

    Site Not Available

  • Peking Union Medical College Hospital

    Beijing 1816670, Beijing Municipality 2038349 100032
    China

    Completed

  • Hospital Pablo Tobon Uribe

    Medellin, Antioquia 050034
    Colombia

    Site Not Available

  • Hospital Pablo Tobon Uribe

    Medellín 3674962, Antioquia 3689815 050034
    Colombia

    Active - Recruiting

  • George Papanikolaou Hospital

    Thessaloniki, 57010
    Greece

    Site Not Available

  • George Papanikolaou Hospital

    Thessaloniki 734077, 57010
    Greece

    Active - Recruiting

  • Semmelweis Egyetem

    Budapest, 1089
    Hungary

    Active - Recruiting

  • Semmelweis University

    Budapest, 1088
    Hungary

    Site Not Available

  • Semmelweis University

    Budapest 3054643, 1083
    Hungary

    Active - Recruiting

  • Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus

    New Delhi, Delhi 110085
    India

    Site Not Available

  • Rajiv Gandhi Cancer Institute and Research Centre

    New Delhi, Delhi 110085
    India

    Active - Recruiting

  • Malabar Cancer Center, Kerala

    Kannur, Kerala 670103
    India

    Site Not Available

  • Malabar Cancer Centre

    Kannur, Kerala 670103
    India

    Site Not Available

  • Amrita Institute of Medical Sciences (AIMS) and Research Centre

    Kochi, Kerala 682041
    India

    Active - Recruiting

  • Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims

    Kochi, Kerala 682041
    India

    Site Not Available

  • Malabar Cancer Center, Kerala

    Kannur 1274987, Kerala 1267254 670103
    India

    Active - Recruiting

  • Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims

    Kochi 1273874, Kerala 1267254 682041
    India

    Active - Recruiting

  • K J Somaiya Super Specialty Hospital & Research Centre

    Mumbai, Maharashtra 400022
    India

    Site Not Available

  • K J Somaiya Super Specialty Hospital & Research Centre

    Mumbai 1275339, Maharashtra 1264418 400022
    India

    Active - Recruiting

  • Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus

    New Delhi 1261481, National Capital Territory of Delhi 1273293 110085
    India

    Active - Recruiting

  • Postgraduate Institute of Medical Education & Research (PGIMER)

    Chandigarh, Punjab 1259223 708248
    India

    Active - Recruiting

  • Bhagwan Mahaveer Cancer Hospital & Research Centre

    Jaipur, Rajasthan 302017
    India

    Active - Recruiting

  • King George Hospital

    Lucknow, Uttar Pradesh 226003
    India

    Site Not Available

  • Kings George's Medical University

    Lucknow, Uttar Pradesh 226014
    India

    Site Not Available

  • King George Hospital

    Lucknow 1264733, Uttar Pradesh 1253626 226003
    India

    Active - Recruiting

  • Sanjay Gandhi Postgraduate Institute of Medical Sciences

    Lucknow 1264733, Uttar Pradesh 1253626 226014
    India

    Active - Recruiting

  • Postgraduate Institute of Medical Education and Research

    Chandigarh, 160012
    India

    Active - Recruiting

  • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)

    Jaipur, 302017
    India

    Site Not Available

  • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)

    Jaipur 1269515, 302017
    India

    Active - Recruiting

  • Malabar Cancer Center, Kerala

    Kerala, 670103
    India

    Site Not Available

  • King George Hospital

    Uttar Pradesh, 226003
    India

    Site Not Available

  • Aou Careggi

    Florence 3176959, Firenze 50139
    Italy

    Active - Recruiting

  • Aou Careggi

    Firenze, Florence 50139
    Italy

    Site Not Available

  • Fondazione Policlinico Universitario A Gemelli

    Roma, Lazio 168
    Italy

    Site Not Available

  • Azienda Ospedaliera Città della Salute e della Scienza di Torino

    Torino, Piemonte 10126
    Italy

    Site Not Available

  • Fondazione Policlinico Universitario A. Gemelli - IRCCS

    Rome 3169070, Roma 00168
    Italy

    Active - Recruiting

  • Fondazione Policlinico Universitrio a. Gemelli - IRCCS

    Rome 3169070, Roma 00168
    Italy

    Active - Recruiting

  • Azienda Ospedaliera Universitaria Careggi

    Firenze, 50134
    Italy

    Active - Recruiting

  • Fondazione Policlinico Universitrio a. Gemelli - IRCCS

    Rome, 000168
    Italy

    Site Not Available

  • Hematology Citta della Salute e della Scienza di Torino

    Turin, 10126
    Italy

    Site Not Available

  • Hematology Citta della Salute e della Scienza di Torino

    Turin 3165524, 10126
    Italy

    Active - Recruiting

  • Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital

    Nagoya 1856057, Aichi-ken 1865694 466-8650
    Japan

    Active - Recruiting

  • Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital

    Nagoya, Aiti 466-8650
    Japan

    Site Not Available

  • Ogaki Municipal Hospital

    Ogaki, Gifu 503-8502
    Japan

    Site Not Available

  • Ogaki Municipal Hospital

    Ogaki city, Gifu 503-8502
    Japan

    Site Not Available

  • Ogaki Municipal Hospital

    Ōgaki 1854703, Gifu 1863640 503-8502
    Japan

    Active - Recruiting

  • University of Tsukuba Hospital

    Tsukuba, Ibaraki 305-8576
    Japan

    Site Not Available

  • University of Tsukuba Hospital

    Tsukuba-shi, Ibaraki 305-8576
    Japan

    Site Not Available

  • University of Tsukuba Hospital

    Tsukuba 2110681, Ibaraki 2112669 305-8576
    Japan

    Active - Recruiting

  • Matsushita Memorial Hospital

    Moriguchi, Osaka 570-8540
    Japan

    Site Not Available

  • Panasonic Health Insurance Organization Matsushita Memorial Hospital

    Moriguchi-city, Osaka 570-8540
    Japan

    Site Not Available

  • Matsushita Memorial Hospital

    Moriguchi 1856584, Osaka 1853904 570-8540
    Japan

    Active - Recruiting

  • NTT Medical Center Tokyo

    Shinagawa-ku, Tokyo 1850144 141-8625
    Japan

    Active - Recruiting

  • Jordan University Hospital (JUH)

    Amman, 11942
    Jordan

    Site Not Available

  • University of Jordan

    Amman, 11196
    Jordan

    Active - Recruiting

  • Jordan University Hospital (JUH)

    Amman 250441, 11942
    Jordan

    Active - Recruiting

  • Ajou University Medical Center

    Suwon, Gyeonggi 16499
    Korea, Republic of

    Site Not Available

  • St. Vincent Hospital

    Suwon-si, Gyeonggi-do 16247
    Korea, Republic of

    Site Not Available

  • Gachon University Gil Medical Center

    Incheon, Namdong-Gu 21565
    Korea, Republic of

    Site Not Available

  • Pusan National University Hospital

    Busan, 49241
    Korea, Republic of

    Site Not Available

  • Ajou University Medical Center

    Gyeonggi-do, 16499
    Korea, Republic of

    Site Not Available

  • Ajou University Medical Center

    Gyeonggido, 16499
    Korea, Republic of

    Site Not Available

  • Ewha Womans University Mokdong Hospital

    Seoul, 07985
    Korea, Republic of

    Active - Recruiting

  • Korea University Hospital

    Seoul, 02841
    Korea, Republic of

    Active - Recruiting

  • Samsung Medical Center

    Seoul, 06351
    Korea, Republic of

    Site Not Available

  • Samsung Medical Center - PPDS

    Seoul, 06351
    Korea, Republic of

    Active - Recruiting

  • Severance Hospital

    Seoul, 03722
    Korea, Republic of

    Active - Recruiting

  • St. Vincent Hospital

    Suwon, 16247
    Korea, Republic of

    Site Not Available

  • Hospital Tengku Ampuan Afzan

    Kuantan, Pahang 25200
    Malaysia

    Active - Recruiting

  • Hospital Tg Ampuan Afzan

    Kuantan, Pahang 25200
    Malaysia

    Site Not Available

  • Hospital Tg Ampuan Afzan

    Kuantan 1735227, Pahang 1733042 25200
    Malaysia

    Active - Recruiting

  • Hospital Queen Elizabeth

    Kota Kinabalu, Sabah 88586
    Malaysia

    Site Not Available

  • Hospital Queen Elizabeth

    Kota Kinabalu 1733432, Sabah 1733039 88586
    Malaysia

    Active - Recruiting

  • Hospital Ampang

    Ampang, Selangor 1733037 68000
    Malaysia

    Active - Recruiting

  • Queen Elizabeth Hospital - Kota Kinabalu

    Kota Kinabalu, 88586
    Malaysia

    Active - Recruiting

  • Servicio de Hematologia del Hospital Universitario de la Uanl

    Monterrey, Nuevo Leon 64460
    Mexico

    Site Not Available

  • Hospital Universitario Dr. Jose Eleuterio González

    Monterrey, Nuevo León 64460
    Mexico

    Active - Recruiting

  • Servicio de Hematologia del Hospital Universitario de la Uanl

    Monterrey 3995465, Nuevo León 3522542 64460
    Mexico

    Active - Recruiting

  • Clinica San Felipe

    Lima, 15072
    Peru

    Site Not Available

  • Clinica San Felipe

    Lima 3936456, 15072
    Peru

    Active - Recruiting

  • St Lukes Medical Center

    Quezon City, Central Luzon 1011
    Philippines

    Site Not Available

  • St Lukes Medical Center

    Quezon 1692199, Central Luzon 7521305 1011
    Philippines

    Active - Recruiting

  • University Clinical Center Medical University of Gdansk

    Gdansk, Pomeranian Voivodeship 80-214
    Poland

    Site Not Available

  • University Clinical Center Medical University of Gdansk

    Gdansk 3099434, Pomeranian Voivodeship 3337496 80-214
    Poland

    Active - Recruiting

  • Klinika Hematologii, Szpital Uniwersytecki Nr 2 im. Jana Biziela w Bydgoszczy

    Bydgoszcz, 85-168
    Poland

    Active - Recruiting

  • Szpital Uniwersytecki Nr2 Bydgoszcz

    Bydgoszcz, 85-168
    Poland

    Site Not Available

  • Szpital Uniwersytecki Nr2 Bydgoszcz

    Bydgoszcz 3102014, 85-168
    Poland

    Active - Recruiting

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-214
    Poland

    Active - Recruiting

  • Institute of Hematology and Transfusion Medicine

    Warsaw, 02-776
    Poland

    Site Not Available

  • Institute of Hematology and Transfusion Medicine

    Warsaw 756135, 02-776
    Poland

    Active - Recruiting

  • Instytut Hematologii i Transfuzjologii

    Warszawa, 02-776
    Poland

    Site Not Available

  • Ion Chiricuta Oncology Institute

    Cluj-Napoca, Cluj 400015
    Romania

    Site Not Available

  • Prof Dr I Chiricuta Institute of Oncology

    Cluj-Napoca, Cluj 400015
    Romania

    Active - Recruiting

  • Ion Chiricuta Oncology Institute

    Cluj-Napoca 681290, Cluj 681291 400015
    Romania

    Active - Recruiting

  • Filantropia Municipal Clinical Hospital

    Craiova, Dolj 200143
    Romania

    Active - Recruiting

  • Municipal Hospital Filantropia

    Craiova, Dolj 200143
    Romania

    Site Not Available

  • Municipal Hospital Filantropia

    Craiova 680332, Dolj 679134 200143
    Romania

    Active - Recruiting

  • Targu Mures Clinical County Emergency Hospital

    Targu Mures, Mures 540136
    Romania

    Site Not Available

  • Targu-Mures Emergency Clinical County Hospital

    Targu Mures, Mures 540136
    Romania

    Active - Recruiting

  • Targu Mures Clinical County Emergency Hospital

    Târgu Mureş 665004, Mureș County 672628 540136
    Romania

    Active - Recruiting

  • National University Hospital

    Singapore, 119074
    Singapore

    Site Not Available

  • National University Hospital

    Singapore 1880252, 119074
    Singapore

    Active - Recruiting

  • Ajou University Medical Center

    Suwon 1835553, Gyeonggi-do 1841610 16499
    South Korea

    Active - Recruiting

  • St. Vincent Hospital

    Suwon 1835553, Gyeonggi-do 1841610 16247
    South Korea

    Active - Recruiting

  • Gachon University Gil Medical Center

    Incheon 1843564, Namdong-Gu 21565
    South Korea

    Active - Recruiting

  • Pusan National University Hospital

    Busan 1838524, 49241
    South Korea

    Active - Recruiting

  • Ewha Womans University Mokdong Hospital

    Seoul 1835848, 07985
    South Korea

    Active - Recruiting

  • Korea University Hospital

    Seoul 1835848, 02841
    South Korea

    Active - Recruiting

  • Samsung Medical Center

    Seoul 1835848, 06351
    South Korea

    Active - Recruiting

  • Seoul St. Mary's Hospital - The Catholic University of Korea

    Seoul 1835848, 065791
    South Korea

    Active - Recruiting

  • Severance Hospital

    Seoul 1835848, 03722
    South Korea

    Active - Recruiting

  • Hospital Universitario Basurto

    Bilbao, Bizkaia 48013
    Spain

    Site Not Available

  • Hospital Universitario Basurto

    Bilbao 3128026, Vizcaya 48013
    Spain

    Active - Recruiting

  • Hospital Clinic de Barcelona

    Barcelona, 08036
    Spain

    Site Not Available

  • Hospital Clinic de Barcelona

    Barcelona 3128760, 08036
    Spain

    Active - Recruiting

  • Hospital General JM Morales Meseguer

    Murcia, 30008
    Spain

    Site Not Available

  • Hospital General JM Morales Meseguer

    Murcia 2513416, 30008
    Spain

    Active - Recruiting

  • Hospital Clinico Universitario de Salamanca

    Salamanca 3111108, 37007
    Spain

    Active - Recruiting

  • China Medical University Hospital

    Taichung, Central Taiwan 40447
    Taiwan

    Site Not Available

  • China Medical University Hospital

    Taichung 1668399, Central Taiwan 40447
    Taiwan

    Active - Recruiting

  • Chang Gung Memorial Hospital - Linkou Branch

    Taoyuan, Hunan Province 33305
    Taiwan

    Site Not Available

  • Chang Gung Memorial Hospital - Linkou Branch

    Taoyuan District 1667905, Hunan Province 33305
    Taiwan

    Active - Recruiting

  • Changhua Christian Hospital

    Changhua 1993459, 500-06
    Taiwan

    Active - Recruiting

  • Changhua Christian Hospital

    Changhua City, 500-06
    Taiwan

    Site Not Available

  • Hualien Tzu Chi Hospital

    Hualien City, 97002
    Taiwan

    Site Not Available

  • Hualien Tzu Chi Hospital

    Hualien City 1674504, 97002
    Taiwan

    Active - Recruiting

  • Kaohsiung Medical University - Chung-Ho Memorial Hospital

    Kaohsiung, 807
    Taiwan

    Active - Recruiting

  • Kaohsiung Medical University Hospital

    Kaohsiung, 80756
    Taiwan

    Site Not Available

  • Kaohsiung Medical University Hospital

    Kaohsiung City 1673820, 80756
    Taiwan

    Active - Recruiting

  • China Medical University Hospital - PPDS

    Taichung, 40447
    Taiwan

    Active - Recruiting

  • Taichung Veterans General Hospital (VGHTC)

    Taichung 1668399, 4070
    Taiwan

    Active - Recruiting

  • Taichung Veterans General Hospital (VGHTC)

    Taichung City, 4070
    Taiwan

    Site Not Available

  • National Cheng Kung University Hospital

    Tainan, 704
    Taiwan

    Site Not Available

  • National Cheng Kung University Hospital

    Tainan 1668355, 704
    Taiwan

    Active - Recruiting

  • National Cheng Kung University Hospital

    Tainan City 1668355, 704
    Taiwan

    Site Not Available

  • National Taiwan University Hospital

    Taipei, 100229
    Taiwan

    Site Not Available

  • Tri-Service General Hospital

    Taipei, 11490
    Taiwan

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei 1668341, 100229
    Taiwan

    Active - Recruiting

  • Tri-Service General Hospital

    Taipei 1668341, 114
    Taiwan

    Active - Recruiting

  • Tri-Service General Hospital

    Taipei City, 114
    Taiwan

    Site Not Available

  • Chang Gung Hospital

    Taoyuan City, 33305
    Taiwan

    Site Not Available

  • National Taiwan University Hospital

    Tapei City, 100229
    Taiwan

    Site Not Available

  • Clinical Research Center, Faculty of Medicine, Prince of Songkla University

    Hat Yai 1610780, Changwat Songkhla 1606146 90110
    Thailand

    Active - Recruiting

  • Rajavithi Hospital

    Ratchathewi, Krung Thep Maha Nakhon-Bangkok 10400
    Thailand

    Site Not Available

  • Clinical Research Center, Faculty of Medicine, Prince of Songkla University

    Hat Yai, Songkhla 90110
    Thailand

    Site Not Available

  • Songklanagarind Hospital Prince of Songkla University

    Hat Yai, Songkhla 90110
    Thailand

    Active - Recruiting

  • King Chulalongkorn Memorial Hospital

    Bangkok, 10330
    Thailand

    Active - Recruiting

  • Rajavithi Hospital

    Bangkok, 10400
    Thailand

    Site Not Available

  • King Chulalongkorn Memorial Hospital

    Bangkok 1609350, 10330
    Thailand

    Active - Recruiting

  • Rajavithi Hospital

    Bangkok 1609350, 10400
    Thailand

    Site Not Available

  • Chaing Mai University

    Chiang Mai, 50200
    Thailand

    Site Not Available

  • Chiang Mai University

    Chiang Mai, 50200
    Thailand

    Active - Recruiting

  • Chaing Mai University

    Chiang Mai 1153671, 50200
    Thailand

    Active - Recruiting

  • Srinagarind Hospital

    Khon Kaen, 4002
    Thailand

    Site Not Available

  • Faculty of Medicine Khon Kaen University

    Khon Kaen 1609776, 40002
    Thailand

    Active - Recruiting

  • Srinagarind Hospital

    Khon Kaen 1609776, 40000
    Thailand

    Active - Recruiting

  • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

    Istanbul, 34418
    Turkey

    Active - Recruiting

  • Istanbul University

    Istanbul, 34418
    Turkey

    Site Not Available

  • Ege Universitesi Tip Fakultesi Hastanesi

    Izmir, 35100
    Turkey

    Active - Recruiting

  • Ege University

    Izmir, 35100
    Turkey

    Site Not Available

  • Istanbul University

    Istanbul 745044, 34418
    Turkey (Türkiye)

    Active - Recruiting

  • Ege University

    Izmir 311046, 35100
    Turkey (Türkiye)

    Active - Recruiting

  • St James Hospital

    Leeds, LS9 7TF
    United Kingdom

    Site Not Available

  • St James University Hospital

    Leeds, LS9 7TF
    United Kingdom

    Active - Recruiting

  • St James Hospital

    Leeds 2644688, LS9 7TF
    United Kingdom

    Active - Recruiting

  • The Oncology Institute of Hope & Innovation

    Whittier, California 90603
    United States

    Site Not Available

  • The Oncology Institute of Hope and Innovation

    Whittier, California 90603
    United States

    Active - Recruiting

  • The Oncology Institute of Hope & Innovation

    Whittier 5409059, California 5332921 90602
    United States

    Active - Recruiting

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