Actively Recruiting
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
Led by Regeneron Pharmaceuticals · Updated on 2026-04-24
35
Participants Needed
8
Research Sites
286 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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)
CONDITIONS
Official Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Paroxysmal Nocturnal Hemoglobinuria (PNH) confirmed by high-sensitivity flow cytometry from prior testing
- Currently treated with marketed eculizumab, ravulizumab, or crovalimab at labeled dose for at least 6 months
- Lactate dehydrogenase (LDH) persistently greater than 1.5 times the upper limit of normal in the previous 6 months attributed to intravascular hemolysis
- At least 2 screening LDH values from different visits as described in the protocol
- Willing and able to comply with clinic or remote visits and study-related procedures
- Completion of full series of meningococcal vaccinations required per protocol and agreement to remain up to date with these vaccinations during the study
You will not qualify if you...
- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants
- Body weight less than 40 kilograms at screening
- Known or suspected C5 mutation refractory to current C5 inhibitor treatment as described in the protocol
- Any active or ongoing infection within 2 weeks of screening or during screening period
- Known hereditary complement deficiency
- Other protocol-defined exclusion criteria apply
AI-Screening
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Trial Site Locations
Total: 8 locations
1
AOU Careggi
Florence, Tuscany, Italy, 50134
Actively Recruiting
2
SC Hematology, AOU Città della Salute e della Scienza di Torino
Torino, Italy, 10126
Actively Recruiting
3
In-Vivo Sp. z o.o.
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland, 85-048
Actively Recruiting
4
Seoul National University Hospital
Seoul, South Korea, 03080
Actively Recruiting
5
Seoul St. Mary's Hospital - The Catholic University of Korea
Seoul, South Korea, 065791
Actively Recruiting
6
Ewha Womans University Mokdong Hospital
Seoul, South Korea, 07985
Actively Recruiting
7
Hospital Universitario Basurto
Bilbao, Vizcaya, Spain, 48013
Actively Recruiting
8
Hospital San Pedro de Alcantara
Cáceres, Spain, 10003
Actively Recruiting
Research Team
C
Clinical Trials Administrator
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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