A Study to Evaluate How Pozelimab + Cemdisiran Combination Therapy Works in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) Whose Current Treatment is Not Working Efficiently
Recruiting now · Phase 3
Conditions studied: Paroxysmal Nocturnal Hemoglobinuria
In brief
This study is researching a treatment combination with two experimental drugs called pozelimab and cemdisiran referred to as "study drugs". Researchers are looking for a better way to treat Paroxysmal Nocturnal Hemoglobinuria (PNH). The aim of the study is to see how well the pozelimab and cemdisiran combination works to lower hemolysis in participants whose PNH has been not well controlled even after taking other complement component 5 (C5) inhibitors, eculizumab/eculizumab biosimilar, ravulizumab or crovalimab. The study is looking at several other research questions, including: * What side effects may happen from taking the study drugs? * How much of the study drugs are in the blood at different times? * Whether the body makes antibodies against the study drug (which could make the study drugs not work as well or could lead to side effects)
Key facts
- Study ID
- NCT07154745
- Run by
- Regeneron Pharmaceuticals
- People needed
- 35
- Starts
- 2026-06-11
- Expected to finish
- 2031-12-04
- Last updated by the study team
- 2026-08-05
Who can join
Age: 18 and older. Sex: any. Healthy volunteers: not accepted.
Where it is running
- Hospital das Clinicas da FMRP USP — Ribeirão Preto, São Paulo, Brazil (enrolling)
- Faculdade de Medicina do ABC — Santo André, São Paulo, Brazil (enrolling)
- Hospital Santa Marcelina — São Paulo, Brazil (enrolling)
- CISSS-CA — Lévis, Quebec, Canada (enrolling)
- Universita Cattolica del Sacro Cuore - Policlinico Universitario A. Gemelli — Rome, Lazio, Italy (enrolling)
- AOU Careggi — Florence, Tuscany, Italy (enrolling)
- SC Hematology, AOU Città della Salute e della Scienza di Torino — Torino, Italy (enrolling)
- In-Vivo Sp. z o.o. — Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland (enrolling)
- Seoul National University Hospital — Seoul, South Korea (enrolling)
- Severance Hospital — Seoul, South Korea (enrolling)
- Seoul St. Mary's Hospital - The Catholic University of Korea — Seoul, South Korea (enrolling)
- Ewha Womans University Mokdong Hospital — Seoul, South Korea (enrolling)
- Samsung Medical Center — Seoul, South Korea (enrolling)
- Hospital Universitario Basurto — Bilbao, Vizcaya, Spain (enrolling)
- Institut Catala d'Oncologia — Barcelona, Spain (enrolling)
- Hospital San Pedro de Alcantara — Cáceres, Spain (enrolling)
- Hospital General JM Morales Meseguer — Murcia, Spain (enrolling)
- Hospital Universitario de Salamanca — Salamanca, Spain (enrolling)
- Istanbul University Istanbul Faculty of Medicine — Istanbul, Turkey (Türkiye) (enrolling)
- Ege University — Izmir, Turkey (Türkiye) (enrolling)
Full record on ClinicalTrials.gov
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