Status:
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
Lead Sponsor:
Regeneron Pharmaceuticals
Conditions:
Paroxysmal Nocturnal Hemoglobinuria
Eligibility:
All Genders
18+ years
Phase:
PHASE3
Brief Summary
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...
Detailed Description
The treatment period has two parts, a Treatment Period (TP, 28 weeks) and an Extension treatment Period (EP, 52 weeks).
Eligibility Criteria
Key Inclusion Criteria:
- Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing
- Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months
- LDH persistently > 1.5 × Upper Limit of Normal (ULN) in the previous 6 months that the Principal Investigator (PI) attributes is due to intravascular hemolysis
- At least 2 screening LDH values from different visits as described in the protocol
- Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol and agreement to continue to remain up to date with these vaccinations during the study
Key Exclusion Criteria:
- Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants
- Body weight <40 kilograms at screening visit
- Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol
- Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol
- Known hereditary complement deficiency
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Key Trial Info
Start Date :
March 31 2026
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
September 23 2031
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT07154745
Start Date
March 31 2026
End Date
September 23 2031
Last Update
April 14 2026
Active Locations (7)
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1
AOU Careggi
Florence, Tuscany, Italy, 50134
2
SC Hematology, AOU Città della Salute e della Scienza di Torino
Torino, Italy, 10126
3
In-Vivo Sp. z o.o.
Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland, 85-048
4
Seoul National University Hospital
Seoul, South Korea, 03080