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: March 4, 2025
Sponsor: Regeneron Pharmaceuticals
Overall Status: Active - Recruiting

Phase

3

Condition

Hemoglobinuria, Paroxysmal

Aplastic Anemia

Red Blood Cell Disorders

Treatment

Eculizumab

Ravulizumab

Cemdisiran

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 patients with paroxysmal nocturnal hemoglobinuria (PNH). The aim of the study is to see how safe and effective the pozelimab + cemdisiran combination is for patients with PNH and how the combination compares with 2 existing treatments, one called ravulizumab and the other called 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 vaccination is less than 2 weeks prior tostudy treatment initiation)

  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: Eculizumab
Phase: 3
Study Start date:
August 01, 2022
Estimated Completion Date:
April 01, 2027

Connect with a study center

  • Toronto General Hospital

    Toronto, Ontario M5G2C4
    Canada

    Active - Recruiting

  • Peking Union Medical College Hospital

    Beijing, Beijing 100032
    China

    Active - Recruiting

  • Hospital Pablo Tobon Uribe

    Medellin, Antioquia 050034
    Colombia

    Active - Recruiting

  • George Papanikolaou Hospital

    Thessaloniki, 57010
    Greece

    Active - Recruiting

  • Semmelweis Egyetem

    Budapest, 1089
    Hungary

    Active - Recruiting

  • Semmelweis University

    Budapest, 1083
    Hungary

    Active - Recruiting

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

    New Delhi, Delhi 110085
    India

    Active - Recruiting

  • Rajiv Gandhi Cancer Institute and Research Centre

    New Delhi, Delhi 110085
    India

    Active - Recruiting

  • Malabar Cancer Center, Kerala

    Kannur, Kerala 670103
    India

    Active - Recruiting

  • 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 855972
    India

    Active - Recruiting

  • K J Somaiya Super Specialty Hospital & Research Centre

    Mumbai, Maharashtra 400022
    India

    Active - Recruiting

  • Postgraduate Institute of Medical Education & Research (PGIMER)

    Chandigarh, Punjab 708248
    India

    Active - Recruiting

  • Bhagwan Mahaveer Cancer Hospital & Research Centre

    Jaipur, Rajasthan 302017
    India

    Active - Recruiting

  • Kings George's Medical University

    Lucknow, Uttar Pradesh 226014
    India

    Site Not Available

  • Postgraduate Institute of Medical Education and Research

    Chandigarh, 160012
    India

    Active - Recruiting

  • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)

    Jaipur, 302017
    India

    Active - Recruiting

  • Malabar Cancer Center, Kerala

    Kerala, 670103
    India

    Site Not Available

  • King George Hospital

    Uttar Pradesh, 226003
    India

    Active - Recruiting

  • Aou Careggi

    Firenze, Forence 50139
    Italy

    Active - Recruiting

  • 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

  • Azienda Ospedaliera Universitaria Careggi

    Firenze, 50134
    Italy

    Active - Recruiting

  • Fondazione Policlinico Universitrio a. Gemelli - IRCCS

    Rome, 000168
    Italy

    Active - Recruiting

  • Hematology Citta della Salute e della Scienza di Torino

    Turin, 10126
    Italy

    Active - Recruiting

  • Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital

    Nagoya, Aiti 466-8650
    Japan

    Active - Recruiting

  • Ogaki Municipal Hospital

    Ogaki, Gifu 503-8502
    Japan

    Active - Recruiting

  • Ogaki Municipal Hospital

    Ogaki city, Gifu 503-8502
    Japan

    Site Not Available

  • University of Tsukuba Hospital

    Tsukuba, Ibaraki 305-8576
    Japan

    Active - Recruiting

  • University of Tsukuba Hospital

    Tsukuba-shi, Ibaraki 305-8576
    Japan

    Site Not Available

  • Matsushita Memorial Hospital

    Moriguchi, Osaka 570-8540
    Japan

    Active - Recruiting

  • Panasonic Health Insurance Organization Matsushita Memorial Hospital

    Moriguchi-city, Osaka 570-8540
    Japan

    Site Not Available

  • NTT Medical Center Tokyo

    Shinagawa-ku, Tokyo 141-8625
    Japan

    Active - Recruiting

  • Jordan University Hospital (JUH)

    Amman, 11942
    Jordan

    Active - Recruiting

  • University of Jordan

    Amman, 11196
    Jordan

    Active - Recruiting

  • St. Vincent Hospital

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

    Active - Recruiting

  • Gachon University Gil Medical Center

    Incheon, Namdong-Gu 21565
    Korea, Republic of

    Active - Recruiting

  • Pusan National University Hospital

    Busan, 49241
    Korea, Republic of

    Active - Recruiting

  • Ajou University Medical Center

    Gyeonggi-do, 16499
    Korea, Republic of

    Site Not Available

  • Ajou University Medical Center

    Gyeonggido, 16499
    Korea, Republic of

    Active - Recruiting

  • 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

    Active - Recruiting

  • 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 Ampang

    Ampang, Pahang 68000
    Malaysia

    Active - Recruiting

  • Hospital Tengku Ampuan Afzan

    Kuantan, Pahang 25200
    Malaysia

    Active - Recruiting

  • Hospital Tg Ampuan Afzan

    Kuantan, Pahang 25100
    Malaysia

    Active - Recruiting

  • Hospital Queen Elizabeth

    Kota Kinabalu, Sabah 88586
    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

    Active - Recruiting

  • Hospital Universitario Dr. Jose Eleuterio González

    Monterrey, Nuevo León 64460
    Mexico

    Active - Recruiting

  • Clinica San Felipe

    Lima, 15072
    Peru

    Active - Recruiting

  • St Lukes Medical Center

    Quezon City, Central Luzon 1011
    Philippines

    Active - Recruiting

  • University Clinical Center Medical University of Gdansk

    Gdansk, Pomeranian Voivodeship 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

    Active - Recruiting

  • Uniwersyteckie Centrum Kliniczne

    Gdansk, 80-214
    Poland

    Active - Recruiting

  • Institute of Hematology and Transfusion Medicine

    Warsaw, 02-776
    Poland

    Active - Recruiting

  • Instytut Hematologii i Transfuzjologii

    Warszawa, 02-776
    Poland

    Site Not Available

  • Ion Chiricuta Oncology Institute

    Cluj-Napoca, Cluj 400124
    Romania

    Active - Recruiting

  • Prof Dr I Chiricuta Institute of Oncology

    Cluj-Napoca, Cluj 400015
    Romania

    Active - Recruiting

  • Filantropia Municipal Clinical Hospital

    Craiova, Dolj 200143
    Romania

    Active - Recruiting

  • Municipal Hospital Filantropia

    Craiova, Dolj 200143
    Romania

    Active - Recruiting

  • Targu Mures Clinical County Emergency Hospital

    Targu Mures, Mures 540136
    Romania

    Active - Recruiting

  • Targu-Mures Emergency Clinical County Hospital

    Targu Mures, Mures 540136
    Romania

    Active - Recruiting

  • National University Hospital

    Singapore, 119074
    Singapore

    Active - Recruiting

  • Hospital Universitario Basurto

    Bilbao, Bizkaia 48013
    Spain

    Active - Recruiting

  • Hospital Clinic de Barcelona

    Barcelona, 08036
    Spain

    Active - Recruiting

  • China Medical University Hospital

    Taichung, Central Taiwan 40447
    Taiwan

    Active - Recruiting

  • Chang Gung Memorial Hospital - Linkou Branch

    Taoyuan, Hunan Province 333
    Taiwan

    Active - Recruiting

  • Changhua Christian Hospital

    Changhua City, 500-06
    Taiwan

    Active - Recruiting

  • Hualien Tzu Chi Hospital

    Hualien City, 97002
    Taiwan

    Active - Recruiting

  • Kaohsiung Medical University - Chung-Ho Memorial Hospital

    Kaohsiung, 807
    Taiwan

    Active - Recruiting

  • Kaohsiung Medical University Hospital

    Kaohsiung, 80756
    Taiwan

    Active - Recruiting

  • China Medical University Hospital - PPDS

    Taichung, 40447
    Taiwan

    Active - Recruiting

  • Taichung Veterans General Hospital (VGHTC)

    Taichung City, 4070
    Taiwan

    Active - Recruiting

  • National Cheng Kung University Hospital

    Tainan, 704
    Taiwan

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei, 100229
    Taiwan

    Active - Recruiting

  • Tri-Service General Hospital

    Taipei, 11490
    Taiwan

    Active - Recruiting

  • Tri-Service General Hospital

    Taipei City, 114
    Taiwan

    Active - Recruiting

  • Chang Gung Hospital

    Taoyuan City, 33305
    Taiwan

    Site Not Available

  • National Taiwan University Hospital

    Tapei City, 100229
    Taiwan

    Site Not Available

  • 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

    Active - Recruiting

  • 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

  • Chaing Mai University

    Chiang Mai, 50200
    Thailand

    Active - Recruiting

  • Chiang Mai University

    Chiang Mai, 50200
    Thailand

    Active - Recruiting

  • Srinagarind Hospital

    Khon Kaen, 4002
    Thailand

    Active - Recruiting

  • Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi

    Istanbul, 34418
    Turkey

    Active - Recruiting

  • Istanbul University

    Istanbul, 34418
    Turkey

    Active - Recruiting

  • Ege Universitesi Tip Fakultesi Hastanesi

    Izmir, 35100
    Turkey

    Active - Recruiting

  • Ege University

    Izmir, 35100
    Turkey

    Active - Recruiting

  • St James Hospital

    Leeds, LS9 7TF
    United Kingdom

    Active - Recruiting

  • St James University Hospital

    Leeds, LS9 7TF
    United Kingdom

    Active - Recruiting

  • The Oncology Institute of Hope & Innovation

    Whittier, California 90603
    United States

    Active - Recruiting

  • The Oncology Institute of Hope and Innovation

    Whittier, California 90603
    United States

    Active - Recruiting

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