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Clinical Trials in China / NCT05133531
Active, not recruiting Phase 3

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

NCT05133531 · tracked via the Priya Life Science China tracker
Sponsor
Regeneron Pharmaceuticals
Phase
Phase 3
Started
2022-08-01
Last updated
2026-04-30

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Investigational drug(s) / intervention(s)

RavulizumabPozelimabCemdisiranEculizumab

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

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

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
Key Inclusion Criteria: 1. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol 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 Key Exclusion Criteria: 1. Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening 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 Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Primary outcome measure(s)

Trial sites (68)

FacilityCityRegionStatus
The Oncology Institute of Hope & Innovation Whittier California
Centro de Estudos e Pesquisas em Hematologia e Oncologia Santo André São Paulo
A Beneficencia Portuguesa de Sao Paulo, BP Mirante São Paulo Brazil
Casa de Saude Santa Marcelina São Paulo Brazil
Toronto General Hospital Toronto Ontario
Peking Union Medical College Hospital Beijing Beijing Municipality
Hospital Pablo Tobon Uribe Medellín Antioquia
George Papanikolaou Hospital Thessaloniki Greece
Semmelweis University Budapest Hungary
Malabar Cancer Center, Kerala Kannur Kerala
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims Kochi Kerala
K J Somaiya Super Specialty Hospital & Research Centre Mumbai Maharashtra
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus New Delhi National Capital Territory of Delhi
Postgraduate Institute of Medical Education & Research (PGIMER) Chandigarh Punjab
King George Hospital Lucknow Uttar Pradesh
Sanjay Gandhi Postgraduate Institute of Medical Sciences Lucknow Uttar Pradesh
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC) Jaipur India
Aou Careggi Florence Firenze
Fondazione Policlinico Universitario A. Gemelli - IRCCS Rome Roma
Hematology Citta della Salute e della Scienza di Torino Turin Italy
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital Nagoya Aichi-ken
Ogaki Municipal Hospital Ōgaki Gifu
University of Tsukuba Hospital Tsukuba Ibaraki
Matsushita Memorial Hospital Moriguchi Osaka
NTT Medical Center Tokyo Shinagawa-ku Tokyo
Jordan University Hospital (JUH) Amman Jordan
Hospital Tg Ampuan Afzan Kuantan Pahang
Hospital Queen Elizabeth Kota Kinabalu Sabah
Hospital Ampang Ampang Selangor
Servicio de Hematologia del Hospital Universitario de la Uanl Monterrey Nuevo León
Clinica San Felipe Lima Peru
St Lukes Medical Center Quezon Central Luzon
University Clinical Center Medical University of Gdansk Gdansk Pomeranian Voivodeship
Szpital Uniwersytecki Nr2 Bydgoszcz Bydgoszcz Poland
Institute of Hematology and Transfusion Medicine Warsaw Poland
Ion Chiricuta Oncology Institute Cluj-Napoca Cluj
Municipal Hospital Filantropia Craiova Dolj
Targu Mures Clinical County Emergency Hospital Târgu Mureş Mureș County
National University Hospital Singapore Singapore
St. Vincent Hospital Suwon Gyeonggi-do

+ 28 more sites — see the full list on the official registry below.

On this site

📄 Ultomiris (ravulizumab) drug profile → 📄 Soliris (eculizumab) drug profile →

More Regeneron Pharmaceuticals trials in China

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05133531 on ClinicalTrials.gov ↗ ← All trials in China