A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works.

Last updated: April 28, 2025
Sponsor: Regeneron Pharmaceuticals
Overall Status: Active - Recruiting

Phase

3

Condition

Aplastic Anemia

White Cell Disorders

Anemia

Treatment

Cemdisiran

Pozelimab

Clinical Study ID

NCT05744921
R3918-PNH-2050
2023-510336-36-00
2021-004931-10
  • Ages > 18
  • All Genders

Study Summary

This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on patients with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for patients with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section.

This study is looking at several other research questions, including:

  • How effective is the pozelimab + cemdisiran combination?

  • What side effects may happen from taking the study drugs?

  • How much of each study drug is in the blood at different times?

  • Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)

Eligibility Criteria

Inclusion

Key Inclusion Criteria:

Patients Entering from the Parent Study

  1. Patients with PNH who have completed, without permanent discontinuation, studytreatment in the parent study (R3918-PNH-2021[NCT05133531]), including thepost-Open-label treatment period (OLTP) transition period, if applicable.

  2. Willing and able to comply with clinic visits and study-related procedures,including meningococcal vaccinations required per protocol.

Patients Entering with C5 polymorphism

  1. Patients with PNH who have a documented C5 polymorphism rendering them refractory toeculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in theprotocol

  2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNHgranulocytes or monocytes

  3. Active disease, as defined by the presence of 1 or more PNH-related sign or symptomas described in the protocol

  4. LDH level ≥2 × upper limit of normal (ULN) at the screening visit

  5. Willing and able to comply with clinic visits and study-related procedures,including meningococcal vaccinations required per protocol

Exclusion

Key Exclusion Criteria:

Patients Entering from the Parent Study

  1. Significant protocol deviation(s) in the parent study based on the investigator'sjudgment and to the extent that these would (if continued) impact the studyobjectives and/or safety of the patient

  2. Any new condition or worsening of an existing condition which, in the opinion of theinvestigator, would make the patient unsuitable for enrollment or could interferewith the patient participating in or completing the study

Patients Entering with C5 polymorphism

  1. Prior treatment with complement inhibitors within 5 half-lives of the respectiveagent prior to screening, except for prior eculizumab or ravulizumab which are notexclusionary

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

  3. Not meeting meningococcal vaccination requirements and, at a minimum, documentationof quadrivalent meningococcal vaccination within 5 years prior to enrollment andserotype B vaccine within 3 years prior to enrollment as described in the protocol

  4. Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA)during screening

  5. Patients with known HIV with history of opportunistic infections in the last 1 yearas described in the protocol

  6. Known hereditary complement deficiency

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

  8. Documented history of liver cirrhosis or patients with liver disease with evidenceof current impaired liver function or patients with elevations in Alanineaminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or itscomplications) as described in the protocol

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Study Design

Total Participants: 202
Treatment Group(s): 2
Primary Treatment: Cemdisiran
Phase: 3
Study Start date:
March 07, 2023
Estimated Completion Date:
February 05, 2029

Connect with a study center

  • Toronto General Hospital

    Toronto, Ontario M5G2 C4
    Canada

    Active - Recruiting

  • Semmelweis University/Semmelweis Egyetem

    Budapest, 1083
    Hungary

    Active - Recruiting

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

    New Delhi, Delhi 110085
    India

    Active - Recruiting

  • Bhagwan Mahaveer Cancer Hospital & Research Centre

    Jaipur, 302017
    India

    Active - Recruiting

  • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)

    Jaipur, 302017
    India

    Active - Recruiting

  • Aou Careggi

    Firenze, Forence 50139
    Italy

    Active - Recruiting

  • Azienda Ospedaliera Careggi

    Firenze, Forence 50139
    Italy

    Active - Recruiting

  • Azienda Ospedaliera Città della Salute e della Scienza di Torino

    Firenze, 50139
    Italy

    Active - Recruiting

  • SC Hematology, AOU Città della Salute e della Scienza di Torino

    Torino, 10126
    Italy

    Active - Recruiting

  • University of Tsukuba Hospital

    Tsukuba, Ibaraki 305-8576
    Japan

    Active - Recruiting

  • St. Vincent Hospital

    Suwon, Gyeonggi 16247
    Korea, Republic of

    Active - Recruiting

  • Ajou University Medical Center

    Suwon-si, Gyeonggi-do 16499
    Korea, Republic of

    Active - Recruiting

  • St. Vincent Hospital

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

    Active - Recruiting

  • Gachon University Gil Medical Center

    Incheon, Namdong-Gu 405-760
    Korea, Republic of

    Active - Recruiting

  • Pusan National University Hospital

    Busan, 49241
    Korea, Republic of

    Active - Recruiting

  • Ewha Womans University Mokdong Hospital

    Seoul, 07985
    Korea, Republic of

    Active - Recruiting

  • Samsung Medical Center

    Seoul, 06351
    Korea, Republic of

    Active - Recruiting

  • Severance Hospital Yonsei University Health System

    Seoul, 03722
    Korea, Republic of

    Active - Recruiting

  • Ajou University Medical Center

    Suwon, 16499
    Korea, Republic of

    Active - Recruiting

  • Hospital Tg Ampuan Afzan

    Kuantan, Pahang 25200
    Malaysia

    Active - Recruiting

  • Hospital Queen Elizabeth

    Kota Kinabalu, Sabah 88200
    Malaysia

    Active - Recruiting

  • Hospital Ampang

    Ampang, Selangor 68000
    Malaysia

    Active - Recruiting

  • St Lukes Medical Center

    Quezon City, National Capital Region 1634
    Philippines

    Active - Recruiting

  • Szpital Uniwersytecki Nr2 Bydgoszcz

    Bydgoszcz, 85-168
    Poland

    Active - Recruiting

  • Prof Dr Ion Chiricuta Cancer Institute

    Cluj Napoca, Cluj 400015
    Romania

    Active - Recruiting

  • Prof Dr Ion Chiricuta Institute of Oncology

    Cluj-Napoca, 400015
    Romania

    Site Not Available

  • 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

  • Hospital Universitario de Basurto

    Bilbao, 48013
    Spain

    Active - Recruiting

  • Chang Gung Memorial Hospital - Linkou Branch

    Taoyuan, Hunan Province 33305
    Taiwan

    Active - Recruiting

  • Hualien Tzu Chi Hospital

    Hualien City, 97002
    Taiwan

    Active - Recruiting

  • China Medical University Hospital

    Taichung, 40447
    Taiwan

    Active - Recruiting

  • National Taiwan University Hospital

    Taipei, 10002
    Taiwan

    Active - Recruiting

  • Tri-Service General Hospital

    Taipei City, 114
    Taiwan

    Active - Recruiting

  • Chang Gung Memorial Hospital- Linkou Branch

    Taoyuan, 333
    Taiwan

    Active - Recruiting

  • Prince Of Songkla Hospital, Prince Of Songkhla University

    Hat-Yai, Songkhla 90110
    Thailand

    Active - Recruiting

  • King Chulalongkorn Memorial Hospital

    Bangkok, 10330
    Thailand

    Active - Recruiting

  • Chiang Mai University

    Chiang Mai, 50200
    Thailand

    Active - Recruiting

  • Songklanagarind Hospital

    Hat Yai, 90110
    Thailand

    Site Not Available

  • Srinagarind Hospital

    Khon Kaen, 40002
    Thailand

    Active - Recruiting

  • Maharaj Nakorn Chiang Mai Hospital

    Muang, 50200
    Thailand

    Site Not Available

  • Ege University Faculty of Medicine

    Bornova, Izmir 35100
    Turkey

    Active - Recruiting

  • Ege Universitesi Tip Fakultesi Hastanesi

    Izmir, 35100
    Turkey

    Site Not Available

  • Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology

    Leeds, LS97TF
    United Kingdom

    Active - Recruiting

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