NCT07154745 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
| NCT ID | NCT07154745 |
| Status | Recruiting |
| Phase | Phase 3 |
| Sponsor | Regeneron Pharmaceuticals |
| Condition | Paroxysmal Nocturnal Hemoglobinuria |
| Study Type | INTERVENTIONAL |
| Enrollment | 35 participants |
| Start Date | 2026-05-30 |
| Primary Completion | 2030-01-06 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
Phase 3 trials are large pivotal studies comparing the treatment to current standard of care or placebo. Your participation directly contributes to the evidence needed for regulatory approval.
This trial targets 35 participants in total. It began in 2026-05-30 with a primary completion date of 2030-01-06.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
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 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)
Eligibility Criteria
Key Inclusion Criteria: 1. Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing 2. Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months 3. 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 4. At least 2 screening LDH values from different visits as described in the protocol 5. 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: 1. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants 2. Body weight \<40 kilograms at screening visit 3. Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol 4. Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol 5. Known hereditary complement deficiency Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Contact & Investigator
Clinical Trial Management
STUDY DIRECTOR
Regeneron Pharmaceuticals
Frequently Asked Questions
Who can join the NCT07154745 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Paroxysmal Nocturnal Hemoglobinuria. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT07154745 trial and what does that mean for participants?
Phase 3 trials are large-scale studies comparing the new treatment to existing standards of care or a placebo. They provide the evidence needed for regulatory approval. This trial targets 35 participants.
Is NCT07154745 currently recruiting?
Yes, NCT07154745 is actively recruiting participants. Contact the research team at clinicaltrials@regeneron.com for enrollment information.
Where is the NCT07154745 trial being conducted?
This trial is being conducted at Ribeirão Preto, Brazil, Florence, Italy, Torino, Italy, Bydgoszcz, Poland and 9 additional locations.
Who is sponsoring the NCT07154745 clinical trial?
NCT07154745 is sponsored by Regeneron Pharmaceuticals. The principal investigator is Clinical Trial Management at Regeneron Pharmaceuticals. The trial plans to enroll 35 participants.