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New drug duo aims to tame rare blood disorder

NCT ID NCT05133531

Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing This study
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study tests a combination of two experimental drugs, pozelimab and cemdisiran, for people with PNH, a rare blood disease that destroys red blood cells. The goal is to see if this combo works better and is safer than current standard treatments (ravulizumab and eculizumab). About 202 participants who have not recently been on complement inhibitors will receive either the new combo or a standard drug, and researchers will track blood markers, transfusion needs, and side effects.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

202 people

The number who actually took part.

Started

Aug 2022

Expected to finish

Oct 2026

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Paroxysmal nocturnal hemoglobinuria

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

More trials for these conditions

Other studies related to the condition(s) this trial covers.