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

A Single Arm Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria With Inadequate Control of Intravascular Hemolysis on Currently Available C5 Inhibitor Therapy

• Status: Recruiting
• Phase: Phase 3
• Study type: Interventional
• Enrollment: 35 (estimated)
• Sponsor: Regeneron Pharmaceuticals · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

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)

Detailed description

The treatment period has two parts, a Treatment Period (TP, 28 weeks) and an Extension treatment Period (EP, 52 weeks).

Conditions

• Paroxysmal Nocturnal Hemoglobinuria

Interventions

Type	Name	Description
DRUG	Pozelimab	Administered per the protocol
DRUG	Cemdisiran	Administered per the protocol

Timeline

• Start date: 2026-03-31
• Primary completion: 2029-11-07
• Completion: 2031-09-23
• First posted: 2025-09-04
• Last updated: 2026-04-14

Locations

7 sites across 4 countries: Italy, Poland, South Korea, Spain

Regulatory

• FDA-regulated drug study