Clinical Trial

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

Study acronym: ACCESS-EXT
Recruiting Phase 3
View on ClinicalTrials.gov →
Summary
This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section. This study is looking at several other research questions, including: * How effective is the pozelimab + cemdisiran combination? * What side effects may happen from taking the study drugs? * How much of each study drug is in the blood at different times? * Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)
Protocol Amendment History 2 changes
notable Primary completion moved earlier: 2029-02-26 -> 2028-10-10 2026-07-31
minor Completion moved earlier: 2029-02-26 -> 2028-10-10 2026-07-31
Trial Details
NCT Number NCT05744921
Lead Sponsor Regeneron Pharmaceuticals
Conditions Paroxysmal Nocturnal Hemoglobinuria
Enrollment 202 participants
Start Date 2023-03-07
Primary Completion 2028-10-10 (estimated)
Study Completion 2028-10-10 (estimated)
Updated on ClinicalTrials.gov 2026-07-30