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
NCT ID: NCT05133531
Last Updated: 2025-11-12
Study Results
The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.
Basic Information
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RECRUITING
PHASE3
190 participants
INTERVENTIONAL
2022-08-01
2027-01-28
Brief Summary
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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)
Detailed Description
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Conditions
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Keywords
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Study Design
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RANDOMIZED
PARALLEL
TREATMENT
NONE
Study Groups
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Cohort A
Randomized 1:1
Ravulizumab
Administered Intravenous (IV) per the protocol
Pozelimab
Administered IV and subcutaneous (SC) per the protocol
Cemdisiran
Administered SC per the protocol
Cohort B
Randomized 1:1
Pozelimab
Administered IV and subcutaneous (SC) per the protocol
Cemdisiran
Administered SC per the protocol
Eculizumab
Administered IV per the protocol
Interventions
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Ravulizumab
Administered Intravenous (IV) per the protocol
Pozelimab
Administered IV and subcutaneous (SC) per the protocol
Cemdisiran
Administered SC per the protocol
Eculizumab
Administered IV per the protocol
Other Intervention Names
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Eligibility Criteria
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Inclusion Criteria
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
Exclusion Criteria
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
18 Years
ALL
No
Sponsors
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Regeneron Pharmaceuticals
INDUSTRY
Responsible Party
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Principal Investigators
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Clinical Trial Management
Role: STUDY_DIRECTOR
Regeneron Pharmaceuticals
Locations
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The Oncology Institute of Hope & Innovation
Whittier, California, United States
Toronto General Hospital
Toronto, Ontario, Canada
Peking Union Medical College Hospital
Beijing, Beijing Municipality, China
Hospital Pablo Tobon Uribe
Medellín, Antioquia, Colombia
George Papanikolaou Hospital
Thessaloniki, , Greece
Semmelweis University
Budapest, , Hungary
Malabar Cancer Center, Kerala
Kannur, Kerala, India
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
Kochi, Kerala, India
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai, Maharashtra, India
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi, National Capital Territory of Delhi, India
Postgraduate Institute of Medical Education & Research (PGIMER)
Chandigarh, Punjab, India
King George Hospital
Lucknow, Uttar Pradesh, India
Sanjay Gandhi Postgraduate Institute of Medical Sciences
Lucknow, Uttar Pradesh, India
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur, , India
Aou Careggi
Florence, Firenze, Italy
Fondazione Policlinico Universitrio a. Gemelli - IRCCS
Rome, Roma, Italy
Hematology Citta della Salute e della Scienza di Torino
Turin, , Italy
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aichi-ken, Japan
Ogaki Municipal Hospital
Ōgaki, Gifu, Japan
University of Tsukuba Hospital
Tsukuba, Ibaraki, Japan
Matsushita Memorial Hospital
Moriguchi, Osaka, Japan
NTT Medical Center Tokyo
Shinagawa-ku, Tokyo, Japan
Jordan University Hospital (JUH)
Amman, , Jordan
Hospital Tg Ampuan Afzan
Kuantan, Pahang, Malaysia
Hospital Queen Elizabeth
Kota Kinabalu, Sabah, Malaysia
Hospital Ampang
Ampang, Selangor, Malaysia
Servicio de Hematologia del Hospital Universitario de la Uanl
Monterrey, Nuevo León, Mexico
Clinica San Felipe
Lima, , Peru
St Lukes Medical Center
Quezon, Central Luzon, Philippines
University Clinical Center Medical University of Gdansk
Gdansk, Pomeranian Voivodeship, Poland
Szpital Uniwersytecki Nr2 Bydgoszcz
Bydgoszcz, , Poland
Institute of Hematology and Transfusion Medicine
Warsaw, , Poland
Ion Chiricuta Oncology Institute
Cluj-Napoca, Cluj, Romania
Municipal Hospital Filantropia
Craiova, Dolj, Romania
Targu Mures Clinical County Emergency Hospital
Târgu Mureş, Mureș County, Romania
National University Hospital
Singapore, , Singapore
St. Vincent Hospital
Suwon, Gyeonggi-do, South Korea
Ajou University Medical Center
Suwon, Gyeonggi-do, South Korea
Gachon University Gil Medical Center
Incheon, Namdong-Gu, South Korea
Pusan National University Hospital
Busan, , South Korea
Korea University Hospital
Seoul, , South Korea
Severance Hospital
Seoul, , South Korea
Samsung Medical Center
Seoul, , South Korea
Ewha Womans University Mokdong Hospital
Seoul, , South Korea
Hospital Universitario Basurto
Bilbao, Vizcaya, Spain
Hospital Clinic de Barcelona
Barcelona, , Spain
Hospital General JM Morales Meseguer
Murcia, , Spain
Hospital Clinico Universitario de Salamanca
Salamanca, , Spain
China Medical University Hospital
Taichung, Central Taiwan, Taiwan
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan District, Hunan Province, Taiwan
Changhua Christian Hospital
Changhua, , Taiwan
Hualien Tzu Chi Hospital
Hualien City, , Taiwan
Kaohsiung Medical University Hospital
Kaohsiung City, , Taiwan
Taichung Veterans General Hospital (VGHTC)
Taichung, , Taiwan
National Cheng Kung University Hospital
Tainan, , Taiwan
National Taiwan University Hospital
Taipei, , Taiwan
Tri-Service General Hospital
Taipei, , Taiwan
Clinical Research Center, Faculty of Medicine, Prince of Songkla University
Hat Yai, Changwat Songkhla, Thailand
King Chulalongkorn Memorial Hospital
Bangkok, , Thailand
Rajavithi Hospital
Bangkok, , Thailand
Chaing Mai University
Chiang Mai, , Thailand
Srinagarind Hospital
Khon Kaen, , Thailand
Istanbul University
Istanbul, , Turkey (Türkiye)
Ege University
Izmir, , Turkey (Türkiye)
St James Hospital
Leeds, , United Kingdom
Countries
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Central Contacts
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Related Links
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Study Informational Website
Other Identifiers
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2020-004486-40
Identifier Type: EUDRACT_NUMBER
Identifier Source: secondary_id
2023-509657-31-00
Identifier Type: REGISTRY
Identifier Source: secondary_id
R3918-PNH-2021
Identifier Type: -
Identifier Source: org_study_id