Clinical Research Directory

Browse clinical research sites, groups, and studies.

Sites Groups Studies

Back to Studies

RECRUITING

NCT05133531

PHASE3

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

Sponsor: Regeneron Pharmaceuticals

View on ClinicalTrials.gov

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)

Official title: A Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

190

Start Date

2022-08-01

Completion Date

2027-01-28

Last Updated

2026-02-24

Healthy Volunteers

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Interventions

DRUG

Ravulizumab

Administered Intravenous (IV) per the protocol

DRUG

Pozelimab

Administered IV and subcutaneous (SC) per the protocol

DRUG

Cemdisiran

Administered SC per the protocol

DRUG

Eculizumab

Administered IV per the protocol

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

Locations (69)

The Oncology Institute of Hope & Innovation

Whittier, California, United States

Centro de Estudos e Pesquisas em Hematologia e Oncologia

Santo André, São Paulo, Brazil

A Beneficencia Portuguesa de Sao Paulo, BP Mirante

São Paulo, Brazil

Casa de Saude Santa Marcelina

São Paulo, Brazil

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 Universitario 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

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

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

Faculty of Medicine Khon Kaen University

Khon Kaen, Thailand

Istanbul University

Istanbul, Turkey (Türkiye)

Ege University

Izmir, Turkey (Türkiye)

St James Hospital

Leeds, United Kingdom

Clinical Research Directory

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

Summary

Key Details

Conditions

Interventions

Eligibility Criteria

Locations (69)