Actively Recruiting
A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy
Led by Regeneron Pharmaceuticals · Updated on 2026-05-26
35
Participants Needed
13
Research Sites
97 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying a combination treatment using two experimental drugs, pozelimab and cemdisiran, for adults with Paroxysmal Nocturnal Hemoglobinuria (PNH) whose condition has not been well controlled by current treatments with complement component 5 (C5) inhibitors like eculizumab, ravulizumab, or crovalimab. The study aims to evaluate how effectively this combination reduces hemolysis and to investigate side effects, blood drug levels, and the body's antibody response to the drugs. The treatment period includes two parts: an initial 28-week Treatment Period (TP) followed by a 52-week Extension Period (EP). Participants receive the combination therapy of pozelimab and cemdisiran administered according to the study protocol. The study monitors the drugs' effects and safety throughout both treatment phases to assess the overall impact on controlling hemolysis. During the study, participants will attend clinic or remote visits for assessments including blood tests to measure lactate dehydrogenase (LDH) levels and hemoglobin, monitoring of fatigue, adverse events, and antibody development against the study drugs. The main outcome measured is the percent change in LDH from baseline to week 28, with ongoing evaluations through week 52. Participants must stay up to date with meningococcal vaccinations and comply with study procedures throughout the trial, which continues until November 2031.
CONDITIONS
Brief 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 confirmed by high-sensitivity flow cytometry from prior testing
- Currently treated with eculizumab, ravulizumab, or crovalimab at labeled dose for at least 6 months
- Lactate dehydrogenase (LDH) persistently greater than 1.5 times upper limit of normal in previous 6 months due to intravascular hemolysis
- At least 2 screening LDH values from different visits
- Willing and able to comply with clinic or remote visits and study procedures
- Completed full series of meningococcal vaccinations and agree to stay up to date during the study
You will not qualify if you...
- Receipt of organ transplant or history of bone marrow or other hematologic transplant
- Body weight less than 40 kilograms at screening
- Known or suspected C5 mutation refractory to current C5 inhibitor treatment
- Any active or ongoing infection within 2 weeks of screening or recent infection as described
- Known hereditary complement deficiency
- Other protocol-defined exclusion criteria may apply
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - 28 weeks
Participants receive combination therapy with Pozelimab and Cemdisiran according to the protocol.
Duration - 52 weeks
Participants who complete the initial treatment period may continue combination therapy in an extension phase.
Trial Site Locations
Total: 13 locations
1
Hospital das Clinicas da FMRP USP
Ribeirão Preto, São Paulo, Brazil, 14051-260
Actively Recruiting
2
AOU Careggi
Florence, Tuscany, Italy, 50134
Actively Recruiting
3
SC Hematology, AOU Città della Salute e della Scienza di Torino
Torino, Italy, 10126
Actively Recruiting
4
In-Vivo Sp. z o.o.
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland, 85-048
Actively Recruiting
5
Seoul National University Hospital
Seoul, South Korea, 03080
Actively Recruiting
6
Seoul St. Mary's Hospital - The Catholic University of Korea
Seoul, South Korea, 065791
Actively Recruiting
7
Ewha Womans University Mokdong Hospital
Seoul, South Korea, 07985
Actively Recruiting
8
Samsung Medical Center
Seoul, South Korea, 6351
Actively Recruiting
9
Hospital Universitario Basurto
Bilbao, Vizcaya, Spain, 48013
Actively Recruiting
10
Hospital San Pedro de Alcantara
Cáceres, Spain, 10003
Actively Recruiting
11
Hospital General JM Morales Meseguer
Murcia, Spain, 30008
Actively Recruiting
12
Hospital Universitario de Salamanca
Salamanca, Spain, 37007
Actively Recruiting
13
Istanbul University Istanbul Faculty of Medicine
Istanbul, Turkey (Türkiye), 34418
Actively Recruiting
Research Team
C
Clinical Trials Administrator
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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