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RECRUITING
NCT07154745
PHASE3

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

Sponsor: Regeneron Pharmaceuticals

View on ClinicalTrials.gov

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)

Official title: 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

Key Details

Gender

All

Age Range

18 Years - Any

Study Type

INTERVENTIONAL

Enrollment

35

Start Date

2026-03-31

Completion Date

2031-09-23

Last Updated

2026-03-19

Healthy Volunteers

No

Interventions

DRUG

Pozelimab

Administered per the protocol

DRUG

Cemdisiran

Administered per the protocol

Locations (5)

AOU Careggi

Florence, Tuscany, Italy

In-Vivo Sp. z o.o.

Bydgoszcz, Kuyavian-Pomeranian Voivodeship, Poland

Seoul National University Hospital

Seoul, South Korea

Ewha Womans University Mokdong Hospital

Seoul, South Korea

Hospital San Pedro de Alcantara

Cáceres, Spain