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New drug combo aims to improve PNH treatment

NCT ID NCT05133531

First seen Nov 01, 2025 · Last updated May 23, 2026 · Updated 28 times

Summary

This study tests a combination of two experimental drugs, pozelimab and cemdisiran, in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The goal is to see if this new combo works better and is safer than current standard treatments (ravulizumab and eculizumab). About 202 participants who have not recently received complement inhibitor therapy will be enrolled. The study measures how well the drugs control red blood cell breakdown and reduce the need for blood transfusions.

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Contacts and locations

Locations

  • A Beneficencia Portuguesa de Sao Paulo, BP Mirante

    São Paulo, 01321-001, Brazil

  • Ajou University Medical Center

    Suwon, Gyeonggi-do, 16499, South Korea

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

    Kochi, Kerala, 682041, India

  • Aou Careggi

    Florence, Firenze, 50139, Italy

  • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)

    Jaipur, 302017, India

  • Casa de Saude Santa Marcelina

    São Paulo, 08270-070, Brazil

  • Centro de Estudos e Pesquisas em Hematologia e Oncologia

    Santo André, São Paulo, 09060-650, Brazil

  • Chaing Mai University

    Chiang Mai, 50200, Thailand

  • Chang Gung Memorial Hospital - Linkou Branch

    Taoyuan, Hunan Province, 33305, Taiwan

  • Changhua Christian Hospital

    Changhua, 500-06, Taiwan

  • China Medical University Hospital

    Taichung, Central Taiwan, 40447, Taiwan

  • Clinica San Felipe

    Lima, 15072, Peru

  • Clinical Research Center, Faculty of Medicine, Prince of Songkla University

    Hat Yai, Changwat Songkhla, 90110, Thailand

  • Ege University

    Izmir, 35100, Turkey (Türkiye)

  • Ewha Womans University Mokdong Hospital

    Seoul, 07985, South Korea

  • Faculty of Medicine Khon Kaen University

    Khon Kaen, 40002, Thailand

  • Fondazione Policlinico Universitario A. Gemelli - IRCCS

    Rome, Roma, 00168, Italy

  • Gachon University Gil Medical Center

    Incheon, Namdong-Gu, 21565, South Korea

  • George Papanikolaou Hospital

    Thessaloniki, 57010, Greece

  • Hematology Citta della Salute e della Scienza di Torino

    Turin, 10126, Italy

  • Hospital Ampang

    Ampang, Selangor, 68000, Malaysia

  • Hospital Clinic de Barcelona

    Barcelona, 08036, Spain

  • Hospital Clinico Universitario de Salamanca

    Salamanca, 37007, Spain

  • Hospital General JM Morales Meseguer

    Murcia, 30008, Spain

  • Hospital Pablo Tobon Uribe

    Medellín, Antioquia, 050034, Colombia

  • Hospital Queen Elizabeth

    Kota Kinabalu, Sabah, 88586, Malaysia

  • Hospital Tg Ampuan Afzan

    Kuantan, Pahang, 25200, Malaysia

  • Hospital Universitario Basurto

    Bilbao, Vizcaya, 48013, Spain

  • Hualien Tzu Chi Hospital

    Hualien City, 97002, Taiwan

  • Institute of Hematology and Transfusion Medicine

    Warsaw, 02-776, Poland

  • Ion Chiricuta Oncology Institute

    Cluj-Napoca, Cluj, 400015, Romania

  • Istanbul University

    Istanbul, 34418, Turkey (Türkiye)

  • Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital

    Nagoya, Aichi-ken, 466-8650, Japan

  • Jordan University Hospital (JUH)

    Amman, 11942, Jordan

  • K J Somaiya Super Specialty Hospital & Research Centre

    Mumbai, Maharashtra, 400022, India

  • Kaohsiung Medical University Hospital

    Kaohsiung City, 80756, Taiwan

  • King Chulalongkorn Memorial Hospital

    Bangkok, 10330, Thailand

  • King George Hospital

    Lucknow, Uttar Pradesh, 226003, India

  • Korea University Hospital

    Seoul, 02841, South Korea

  • Malabar Cancer Center, Kerala

    Kannur, Kerala, 670103, India

  • Matsushita Memorial Hospital

    Moriguchi, Osaka, 570-8540, Japan

  • Municipal Hospital Filantropia

    Craiova, Dolj, 200143, Romania

  • NTT Medical Center Tokyo

    Shinagawa-ku, Tokyo, 141-8625, Japan

  • National Cheng Kung University Hospital

    Tainan, 704, Taiwan

  • National Taiwan University Hospital

    Taipei, 100229, Taiwan

  • National University Hospital

    Singapore, 119074, Singapore

  • Ogaki Municipal Hospital

    Ōgaki, Gifu, 503-8502, Japan

  • Peking Union Medical College Hospital

    Beijing, Beijing Municipality, 100032, China

  • Postgraduate Institute of Medical Education & Research (PGIMER)

    Chandigarh, Punjab, 708248, India

  • Pusan National University Hospital

    Busan, 49241, South Korea

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

    New Delhi, National Capital Territory of Delhi, 110085, India

  • Samsung Medical Center

    Seoul, 06351, South Korea

  • Sanjay Gandhi Postgraduate Institute of Medical Sciences

    Lucknow, Uttar Pradesh, 226014, India

  • Semmelweis University

    Budapest, 1083, Hungary

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

    Seoul, 065791, South Korea

  • Servicio de Hematologia del Hospital Universitario de la Uanl

    Monterrey, Nuevo León, 64460, Mexico

  • Severance Hospital

    Seoul, 03722, South Korea

  • St James Hospital

    Leeds, LS9 7TF, United Kingdom

  • St Lukes Medical Center

    Quezon, Central Luzon, 1011, Philippines

  • St. Vincent Hospital

    Suwon, Gyeonggi-do, 16247, South Korea

  • Szpital Uniwersytecki Nr2 Bydgoszcz

    Bydgoszcz, 85-168, Poland

  • Taichung Veterans General Hospital (VGHTC)

    Taichung, 4070, Taiwan

  • Targu Mures Clinical County Emergency Hospital

    Târgu Mureş, Mureș County, 540136, Romania

  • The Oncology Institute of Hope & Innovation

    Whittier, California, 90602, United States

  • Toronto General Hospital

    Toronto, Ontario, M5G 2C4, Canada

  • Tri-Service General Hospital

    Taipei, 114, Taiwan

  • University Clinical Center Medical University of Gdansk

    Gdansk, Pomeranian Voivodeship, 80-214, Poland

  • University of Tsukuba Hospital

    Tsukuba, Ibaraki, 305-8576, Japan

Conditions

Explore the condition pages connected to this study.