Actively Recruiting
Long-Term Safety and Efficacy Study of Pozelimab and Cemdisiran Combination Therapy in Adults With Paroxysmal Nocturnal Hemoglobinuria
Led by Regeneron Pharmaceuticals · Updated on 2026-03-18
202
Participants Needed
43
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are studying an experimental combination of two drugs, pozelimab and cemdisiran, to evaluate their long-term safety and effectiveness in adults with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder. The study aims to understand how well this combination controls the disease, what side effects may occur, how the drugs behave in the body, and whether the body develops antibodies against them. The study includes two groups: patients who completed treatment in a previous related study and patients with PNH who have a specific genetic variation making them resistant to other treatments. Participants receive the combination therapy, with a loading dose of pozelimab given intravenously on the first day. The study is open-label and non-randomized, focusing on long-term treatment effects and safety. Participants will attend regular clinic visits for up to 108 weeks, where researchers will monitor adverse events, blood levels of the drugs, disease activity markers like lactate dehydrogenase (LDH), and symptom changes. They will also track transfusion needs, hemoglobin stability, fatigue, physical function, and quality of life using questionnaires. Safety assessments include monitoring serious adverse events and immune responses to the drugs throughout the study period.
CONDITIONS
Brief Title
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
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients with PNH who completed treatment without permanent discontinuation in the parent study (R3918-PNH-2021), including transition periods
- Patients with PNH having a documented C5 polymorphism making them resistant to eculizumab or ravulizumab
- Diagnosis of PNH confirmed by high-sensitivity flow cytometry with PNH granulocytes or monocytes
- Active PNH disease with at least one related sign or symptom
- Lactate dehydrogenase (LDH) level at least 2 times the upper limit of normal at screening
- Willing and able to comply with clinic visits and required meningococcal vaccinations as per protocol
You will not qualify if you...
- Significant protocol deviations in the parent study impacting safety or study objectives
- New or worsening health conditions making participation unsuitable or unsafe
- Prior treatment with complement inhibitors (except eculizumab or ravulizumab) within 5 half-lives before screening
- History of organ, bone marrow, or hematologic transplant
- Not meeting meningococcal vaccination requirements including recent quadrivalent and serotype B vaccines
- Positive hepatitis B surface antigen or hepatitis C virus RNA at screening
- Known HIV with opportunistic infections within the past year
- Known hereditary complement deficiency
- Active, uncontrolled systemic autoimmune diseases
- History of liver cirrhosis or liver disease with impaired function or elevated liver enzymes unrelated to PNH
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.
1 visit (in-person)
Duration - Up to 108 weeks
Participants receive long-term treatment with the combination of Pozelimab and Cemdisiran to evaluate safety and efficacy in managing Paroxysmal Nocturnal Hemoglobinuria (PNH).
Regular visits throughout up to 108 weeks for treatment administration and monitoring
Trial Site Locations
Total: 43 locations
1
Toronto General Hospital
Toronto, Ontario, Canada, M5G 2C4
Actively Recruiting
2
Hospital Pablo Tobon Uribe
Medellín, Antioquia, Colombia, 050034
Actively Recruiting
3
Semmelweis University/Semmelweis Egyetem
Budapest, Hungary, 1083
Actively Recruiting
4
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
Kochi, Kerala, India, 682041
Actively Recruiting
5
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai, Maharashtra, India, 400022
Actively Recruiting
6
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi, National Capital Territory of Delhi, India, 110085
Actively Recruiting
7
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur, India, 302017
Actively Recruiting
8
Aou Careggi
Florence, Firenze, Italy, 50139
Actively Recruiting
9
SC Hematology, AOU Città della Salute e della Scienza di Torino
Torino, Italy, 10126
Actively Recruiting
10
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aichi-ken, Japan, 466-8650
Withdrawn
11
University of Tsukuba Hospital
Tsukuba, Ibaraki, Japan, 305-8576
Actively Recruiting
12
Jordan University Hospital (JUH)
Amman, Jordan, 11942
Actively Recruiting
13
Hospital Tg Ampuan Afzan
Kuantan, Pahang, Malaysia, 25200
Actively Recruiting
14
Hospital Queen Elizabeth
Kota Kinabalu, Sabah, Malaysia, 88200
Actively Recruiting
15
Hospital Ampang
Ampang, Selangor, Malaysia, 68000
Actively Recruiting
16
Clinica San Felipe
Lima, Peru, 15072
Actively Recruiting
17
St Lukes Medical Center
Quezon City, National Capital Region, Philippines, 1634
Actively Recruiting
18
In-Vivo Sp. z o.o.
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland, 85-048
Actively Recruiting
19
University Clinical Center Medical University of Gdansk
Gdansk, Pomeranian Voivodeship, Poland, 80-214
Actively Recruiting
20
Szpital Uniwersytecki Nr2 Bydgoszcz
Bydgoszcz, Poland, 85-168
Actively Recruiting
21
Prof Dr Ion Chiricuta Cancer Institute
Cluj-Napoca, Cluj, Romania, 400015
Actively Recruiting
22
National University Hospital
Singapore, Singapore, 119074
Actively Recruiting
23
St. Vincent Hospital
Suwon, Gyeonggi-do, South Korea, 16247
Actively Recruiting
24
Ajou University Medical Center
Suwon, Gyeonggi-do, South Korea, 16499
Actively Recruiting
25
Gachon University Gil Medical Center
Incheon, Namdong-Gu, South Korea, 21565
Actively Recruiting
26
Pusan National University Hospital
Busan, South Korea, 49241
Actively Recruiting
27
Severance Hospital Yonsei University Health System
Seoul, South Korea, 03722
Actively Recruiting
28
Samsung Medical Center
Seoul, South Korea, 06351
Actively Recruiting
29
Ewha Womans University Mokdong Hospital
Seoul, South Korea, 07985
Actively Recruiting
30
Hospital Universitario Basurto
Bilbao, Vizcaya, Spain, 48013
Actively Recruiting
31
Hospital Clinic de Barcelona
Barcelona, Spain, 08036
Actively Recruiting
32
China Medical University Hospital
Taichung, Central Taiwan, Taiwan, 40447
Actively Recruiting
33
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan, Hunan Province, Taiwan, 33305
Actively Recruiting
34
Hualien Tzu Chi Hospital
Hualien City, Taiwan, 97002
Actively Recruiting
35
National Taiwan University Hospital
Taipei, Taiwan, 10002
Actively Recruiting
36
Tri-Service General Hospital
Taipei, Taiwan, 11490
Actively Recruiting
37
Prince Of Songkla Hospital, Prince Of Songkhla University
Hat Yai, Changwat Songkhla, Thailand, 90110
Actively Recruiting
38
King Chulalongkorn Memorial Hospital
Bangkok, Thailand, 10330
Actively Recruiting
39
Chiang Mai University
Chiang Mai, Thailand, 50200
Actively Recruiting
40
Faculty of Medicine Khon Kaen University
Khon Kaen, Thailand, 40002
Actively Recruiting
41
Ege University Faculty of Medicine
Bornova, İzmir, Turkey (Türkiye), 35100
Actively Recruiting
42
Istanbul University
Istanbul, Turkey (Türkiye), 34418
Actively Recruiting
43
Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology
Leeds, United Kingdom, LS97TF
Actively Recruiting
Research Team
C
Clinical Trials Administrator
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NON_RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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