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New drug combo aims to control rare blood disease Long-Term

NCT ID NCT05744921

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 study
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
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 25, 2026 · Last updated Sep 18, 2026 · Updated 7 times

Summary

This study is testing the long-term safety and effectiveness of two experimental drugs, pozelimab and cemdisiran, taken together for paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. About 202 participants who completed a prior study will continue treatment to see how well the combination controls red blood cell destruction and reduces the need for transfusions. The focus is on side effects and how the body responds over time.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
pozelimab and cemdisiran
What this could lead to
If successful, this combination could offer a new long-term treatment option for people with PNH, helping control the disease and reduce the need for blood transfusions.
What could go wrong
This is an extension study, so the drugs have already shown some promise, but long-term safety and effectiveness are still being evaluated. Side effects or the body developing antibodies could limit benefits.

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

About 202 people

The number the study aims to enrol. It can still change while the study runs.

Started

Mar 2023

Expected to finish

Oct 2028

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: Patients Entering from the Parent Study 1. Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021\[NCT05133531\]), including the post-Open-label treatment period (OLTP) transition period, if applicable. 2. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol. Patients Entering with C5 polymorphism 1. Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol 2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes 3. Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol 4. LDH level ≥2 × upper limit of normal (ULN) at the screening visit 5. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol Key Exclusion Criteria: Patients Entering from the Parent Study 1. Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient 2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study Patients Entering with C5 polymorphism 1. Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary 2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant 3. Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype B vaccine within 3 years prior to enrollment as described in the protocol 4. Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening 5. Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol 6. Known hereditary complement deficiency 7. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases 8. Documented history of liver cirrhosis or patients with liver disease with evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol 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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    45 sites in 19 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Ajou University Medical Center

    RECRUITING

    Suwon, Gyeonggi-do, 16499, South Korea

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

    RECRUITING

    Kochi, Kerala, 682041, India

  • Aou Careggi

    RECRUITING

    Florence, Firenze, 50139, Italy

  • Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)

    RECRUITING

    Jaipur, 302017, India

  • Chang Gung Memorial Hospital - Linkou Branch

    RECRUITING

    Taoyuan, Hunan Province, 33305, Taiwan

  • Changhua Christian Hospital

    RECRUITING

    Changhua, 500, Taiwan

  • Chiang Mai University

    RECRUITING

    Chiang Mai, 50200, Thailand

  • China Medical University Hospital

    RECRUITING

    Taichung, Central Taiwan, 40447, Taiwan

  • Clinica San Felipe

    RECRUITING

    Lima, 15072, Peru

  • Ege University Faculty of Medicine

    RECRUITING

    Bornova, İzmir, 35100, Turkey (Türkiye)

  • Ewha Womans University Mokdong Hospital

    RECRUITING

    Seoul, 07985, South Korea

  • Faculty of Medicine Khon Kaen University

    RECRUITING

    Khon Kaen, 40002, Thailand

  • Gachon University Gil Medical Center

    RECRUITING

    Incheon, Namdong-Gu, 21565, South Korea

  • Hospital Ampang

    RECRUITING

    Ampang, Selangor, 68000, Malaysia

  • Hospital Clinic de Barcelona

    RECRUITING

    Barcelona, 08036, Spain

  • Hospital Pablo Tobon Uribe

    RECRUITING

    Medellín, Antioquia, 050034, Colombia

  • Hospital Queen Elizabeth

    RECRUITING

    Kota Kinabalu, Sabah, 88200, Malaysia

  • Hospital Tg Ampuan Afzan

    RECRUITING

    Kuantan, Pahang, 25200, Malaysia

  • Hospital Universitario Basurto

    RECRUITING

    Bilbao, Vizcaya, 48013, Spain

  • Hualien Tzu Chi Hospital

    RECRUITING

    Hualien City, 97002, Taiwan

  • In-Vivo Sp. z o.o.

    RECRUITING

    Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-048, Poland

  • Istanbul University

    RECRUITING

    Istanbul, 34418, Turkey (Türkiye)

  • Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital

    WITHDRAWN

    Nagoya, Aichi-ken, 466-8650, Japan

  • Jordan University Hospital (JUH)

    RECRUITING

    Amman, 11942, Jordan

  • K J Somaiya Super Specialty Hospital & Research Centre

    RECRUITING

    Mumbai, Maharashtra, 400022, India

  • King Chulalongkorn Memorial Hospital

    RECRUITING

    Bangkok, 10330, Thailand

  • Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology

    RECRUITING

    Leeds, LS97TF, United Kingdom

  • National Taiwan University Hospital

    RECRUITING

    Taipei, 10002, Taiwan

  • National University Hospital

    RECRUITING

    Singapore, 119074, Singapore

  • Postgraduate Institute of Medical Education & Research

    RECRUITING

    Chandigarh, 160012, India

  • Prince Of Songkla Hospital, Prince Of Songkhla University

    RECRUITING

    Hat Yai, Changwat Songkhla, 90110, Thailand

  • Prof Dr Ion Chiricuta Cancer Institute

    RECRUITING

    Cluj-Napoca, Cluj, 400015, Romania

  • Pusan National University Hospital

    RECRUITING

    Busan, 49241, South Korea

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

    RECRUITING

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

  • SC Hematology, AOU Città della Salute e della Scienza di Torino

    RECRUITING

    Torino, 10126, Italy

  • Samsung Medical Center

    RECRUITING

    Seoul, 06351, South Korea

  • Semmelweis University/Semmelweis Egyetem

    RECRUITING

    Budapest, 1083, Hungary

  • Severance Hospital Yonsei University Health System

    RECRUITING

    Seoul, 03722, South Korea

  • St Lukes Medical Center

    RECRUITING

    Quezon City, National Capital Region, 1634, Philippines

  • St. Vincent Hospital

    RECRUITING

    Suwon, Gyeonggi-do, 16247, South Korea

  • Szpital Uniwersytecki Nr2 Bydgoszcz

    COMPLETED

    Bydgoszcz, 85-168, Poland

  • Taichung Veterans General Hospital (VGHTC)

    RECRUITING

    Taichung, 4070, Taiwan

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

    RECRUITING

    Seoul, 06591, South Korea

  • Toronto General Hospital

    RECRUITING

    Toronto, Ontario, M5G 2C4, Canada

  • Tri-Service General Hospital

    RECRUITING

    Taipei, 11490, Taiwan

  • University Clinical Center Medical University of Gdansk

    RECRUITING

    Gdansk, Pomeranian Voivodeship, 80-214, Poland

  • University of Tsukuba Hospital

    RECRUITING

    Tsukuba, Ibaraki, 305-8576, Japan

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