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

NCT ID NCT05744921

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

First seen Jun 25, 2026 · Last updated Jun 27, 2026 · Updated 1 time

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.

Disclaimer Read more

This is a summary of the original study . Summaries may miss details or leave out important information. Before applying or accepting participation, make sure you have read and understood the full study. Curemydisease.com takes no responsibility whatsoever for anything missed, misunderstood, or acted upon as a result of our summary — we know it does not capture everything.

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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

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

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
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
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
RECRUITING

Bydgoszcz, 85-168, Poland
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