Status:
NOT_YET_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
Inclusion
- Key
- 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
- 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 :
December 19 2025
Trial Type :
INTERVENTIONAL
Allocation :
ESTIMATED
End Date :
June 13 2031
Estimated Enrollment :
35 Patients enrolled
Trial Details
Trial ID
NCT07154745
Start Date
December 19 2025
End Date
June 13 2031
Last Update
November 13 2025
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