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Study identifier: NCT05744921 Synced from ClinicalTrials.gov · July 28, 2026
● Recruiting

A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works

Condition: Paroxysmal Nocturnal Hemoglobinuria  ·  Sponsor: Regeneron Pharmaceuticals

PhasePhase 3
Planned participants202
Who can joinAll sexes, 18 Years to no upper limit
Healthy volunteersNo

About this study

This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section. This study is looking at several other research questions, including: * How effective is the pozelimab + cemdisiran combination? * What side effects may happen from taking the study drugs? * How much of each study drug is in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)

This description comes directly from the study's public registry record.

Talk to the study team

Clinical Trials Administrator  ·  844-734-6643  ·  clinicaltrials@regeneron.com

Always discuss trial participation with your own doctor first.

Locations (45)

Toronto General HospitalToronto, Ontario, CanadaRecruiting
Hospital Pablo Tobon UribeMedellín, Antioquia, ColombiaRecruiting
Semmelweis University/Semmelweis EgyetemBudapest, HungaryRecruiting
Amrita Institute of Medical Sciences (AIMS) and Research Centre AimsKochi, Kerala, IndiaRecruiting
K J Somaiya Super Specialty Hospital & Research CentreMumbai, Maharashtra, IndiaRecruiting
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini CampusNew Delhi, National Capital Territory of Delhi, IndiaRecruiting
Postgraduate Institute of Medical Education & ResearchChandigarh, IndiaRecruiting
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)Jaipur, IndiaRecruiting
Aou CareggiFlorence, Firenze, ItalyRecruiting
SC Hematology, AOU Città della Salute e della Scienza di TorinoTorino, ItalyRecruiting
Japanese Red Cross Aichi Medical Center Nagoya Daini HospitalNagoya, Aichi-ken, JapanWithdrawn
University of Tsukuba HospitalTsukuba, Ibaraki, JapanRecruiting

+ 33 more locations — full list on the registry record.

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Source record: clinicaltrials.gov/study/NCT05744921