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New drug duo aims to rescue PNH patients failing standard care

NCT ID NCT07154745

Recruiting now Disease control Sponsor: Regeneron Pharmaceuticals Source: ClinicalTrials.gov ↗

First seen Jun 27, 2026 · Last updated Aug 06, 2026 · Updated 5 times

Summary

This study tests two experimental drugs, pozelimab and cemdisiran, together in 35 adults with paroxysmal nocturnal hemoglobinuria (PNH) whose red blood cell destruction is not well-controlled by current C5 inhibitors. The goal is to see if the combination can lower hemolysis (breakdown of red blood cells) better than existing treatments. Participants will receive the drugs by injection and be monitored for side effects and blood levels.

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Conditions

The condition(s) this trial relates to.

Hemoglobinuria, Paroxysmal paroxysmal nocturnal hemoglobinuria

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

PAROXYSMAL NOCTURNAL HEMOGLOBINURIA

Contacts and locations

Locations

  • AOU Careggi

    RECRUITING

    Florence, Tuscany, 50134, Italy

  • CISSS-CA

    RECRUITING

    Lévis, Quebec, G6V 3Z1, Canada

  • Ege University

    RECRUITING

    Izmir, 35100, Turkey (Türkiye)

  • Ewha Womans University Mokdong Hospital

    RECRUITING

    Seoul, 07985, South Korea

  • Faculdade de Medicina do ABC

    RECRUITING

    Santo André, São Paulo, 09060 870, Brazil

  • Hospital General JM Morales Meseguer

    RECRUITING

    Murcia, 30008, Spain

  • Hospital San Pedro de Alcantara

    RECRUITING

    Cáceres, 10003, Spain

  • Hospital Santa Marcelina

    RECRUITING

    São Paulo, 08270-070, Brazil

  • Hospital Universitario Basurto

    RECRUITING

    Bilbao, Vizcaya, 48013, Spain

  • Hospital Universitario de Salamanca

    RECRUITING

    Salamanca, 37007, Spain

  • Hospital das Clinicas da FMRP USP

    RECRUITING

    Ribeirão Preto, São Paulo, 14051-260, Brazil

  • In-Vivo Sp. z o.o.

    RECRUITING

    Bydgoszcz, Kuyavian-Pomeranian Voivodeship, 85-048, Poland

  • Institut Catala d'Oncologia

    RECRUITING

    Barcelona, 08908, Spain

  • Istanbul University Istanbul Faculty of Medicine

    RECRUITING

    Istanbul, 34418, Turkey (Türkiye)

  • SC Hematology, AOU Città della Salute e della Scienza di Torino

    RECRUITING

    Torino, 10126, Italy

  • Samsung Medical Center

    RECRUITING

    Seoul, 6351, South Korea

  • Seoul National University Hospital

    RECRUITING

    Seoul, 03080, South Korea

  • Seoul St. Mary's Hospital - The Catholic University of Korea

    RECRUITING

    Seoul, 065791, South Korea

  • Severance Hospital

    RECRUITING

    Seoul, 03722, South Korea

  • Universita Cattolica del Sacro Cuore - Policlinico Universitario A. Gemelli

    RECRUITING

    Rome, Lazio, 00168, Italy

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