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New drug duo aims to rescue PNH patients failing standard care

NCT ID NCT07154745

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

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Phase 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

About 35 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jun 2026

Expected to finish

Dec 2031

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

18 years and older

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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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

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    20 sites in 7 countries. The list below names each one and where it is.

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

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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