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NCT05744921From ClinicalTrials.govRecruiting

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

An Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria

  • Paroxysmal Nocturnal Hemoglobinuria

At a glance

Phase
Phase 3
Study type
Interventional
Sponsor
Regeneron Pharmaceuticals
Enrolment target
202
Started
7 March 2023
Main results due
10 October 2028
Study sites
48
Registry updated
28 September 2026

Can you take part?

  • Aged 18 years and over.
  • Open to any sex.
  • You need the condition being studied — healthy volunteers are not accepted.

These are the headline rules only. Every study has a longer list, and whether you are eligible is decided by the research team at the site — never by this page.

Read the full eligibility criteria
Key Inclusion Criteria:

Patients Entering from the Parent Study

1. Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021\[NCT05133531\]), including the post-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 to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol
2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes
3. Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as 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

Key Exclusion Criteria:

Patients Entering from the Parent Study

1. Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient
2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with 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 respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary
2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
3. Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype 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 year as 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 evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol

Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

What this study is about

In the sponsor’s own words, from the registry.

This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people 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)

Study sites(48)

  • A Beneficencia Portuguesa de Sao Paulo, BP Mirante

    São Paulo, Brazil

    Recruiting

  • Toronto General Hospital

    Toronto, Ontario, Canada

    Recruiting

  • Hospital Pablo Tobon Uribe

    Medellín, Antioquia, Colombia

    Recruiting

  • Semmelweis University/Semmelweis Egyetem

    Budapest, Hungary

    Recruiting

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

    Kochi, Kerala, India

    Recruiting

  • K J Somaiya Super Specialty Hospital & Research Centre

    Mumbai, Maharashtra, India

    Recruiting

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

    New Delhi, National Capital Territory of Delhi, India

    Recruiting

  • Postgraduate Institute of Medical Education & Research

    Chandigarh, India

    Recruiting

  • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)

    Jaipur, India

    Recruiting

  • Aou Careggi

    Florence, Firenze, Italy

    Recruiting

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

    Torino, Italy

    Recruiting

  • Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital

    Nagoya, Aichi-ken, Japan

    Withdrawn

  • University of Tsukuba Hospital

    Tsukuba, Ibaraki, Japan

    Recruiting

  • Jordan University Hospital (JUH)

    Amman, Jordan

    Recruiting

  • Hospital Tg Ampuan Afzan

    Kuantan, Pahang, Malaysia

    Recruiting

  • Hospital Queen Elizabeth

    Kota Kinabalu, Sabah, Malaysia

    Recruiting

  • Hospital Ampang

    Ampang, Selangor, Malaysia

    Recruiting

  • Clinica San Felipe

    Lima, Peru

    Recruiting

  • St Lukes Medical Center

    Quezon City, National Capital Region, Philippines

    Recruiting

  • In-Vivo Sp. z o.o.

    Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland

    Recruiting

  • University Clinical Center Medical University of Gdansk

    Gdansk, Pomeranian Voivodeship, Poland

    Recruiting

  • Szpital Uniwersytecki Nr2 Bydgoszcz

    Bydgoszcz, Poland

    Completed

  • Prof Dr Ion Chiricuta Cancer Institute

    Cluj-Napoca, Cluj, Romania

    Recruiting

  • National University Hospital

    Singapore, Singapore

    Recruiting

  • St. Vincent Hospital

    Suwon, Gyeonggi-do, South Korea

    Recruiting

  • Ajou University Medical Center

    Suwon, Gyeonggi-do, South Korea

    Recruiting

  • Gachon University Gil Medical Center

    Incheon, Namdong-Gu, South Korea

    Recruiting

  • Pusan National University Hospital

    Busan, South Korea

    Recruiting

  • Severance Hospital Yonsei University Health System

    Seoul, South Korea

    Recruiting

  • Samsung Medical Center

    Seoul, South Korea

    Recruiting

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

    Seoul, South Korea

    Recruiting

  • Ewha Womans University Mokdong Hospital

    Seoul, South Korea

    Recruiting

  • Hospital Universitario Basurto

    Bilbao, Vizcaya, Spain

    Recruiting

  • Hospital Clinic de Barcelona

    Barcelona, Spain

    Recruiting

  • China Medical University Hospital

    Taichung, Central Taiwan, Taiwan

    Recruiting

  • Chang Gung Memorial Hospital - Linkou Branch

    Taoyuan, Hunan Province, Taiwan

    Recruiting

  • Changhua Christian Hospital

    Changhua, Taiwan

    Recruiting

  • Hualien Tzu Chi Hospital

    Hualien City, Taiwan

    Recruiting

  • Taichung Veterans General Hospital (VGHTC)

    Taichung, Taiwan

    Recruiting

  • National Taiwan University Hospital

    Taipei, Taiwan

    Recruiting

  • Tri-Service General Hospital

    Taipei, Taiwan

    Recruiting

  • Prince Of Songkla Hospital, Prince Of Songkhla University

    Hat Yai, Changwat Songkhla, Thailand

    Recruiting

  • King Chulalongkorn Memorial Hospital

    Bangkok, Thailand

    Recruiting

  • Chiang Mai University

    Chiang Mai, Thailand

    Recruiting

  • Faculty of Medicine Khon Kaen University

    Khon Kaen, Thailand

    Recruiting

  • Ege University Faculty of Medicine

    Bornova, İzmir, Turkey (Türkiye)

    Recruiting

  • Istanbul University

    Istanbul, Turkey (Türkiye)

    Recruiting

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

    Leeds, United Kingdom

    Recruiting

Showing 48 of 48 sites. 46 of the 48 sites on this study are recruiting right now — a site can stop enrolling while the study as a whole is still open.

Source: ClinicalTrials.gov record NCT05744921. Trialion does not run this study, is not paid to refer anyone to it, and cannot enrol you. Eligibility is always decided by the research team at the site.

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A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatm | Trialion