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

NCT07154745 | Recruiting Phase 3 FDA Drug

• 2 other identifiers: R3918-PNH-24832024-519709-37-00
• Study Type: interventional
• Target: 35
• Locations: 7 countries
• Sites: 19

Brief 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)

Conditions

Paroxysmal Nocturnal Hemoglobinuria

Keywords

PNH

Outcome Measures

Primary Outcomes (1)

• Percent change in Lactate Dehydrogenase (LDH) during TP

  From baseline to week 28

Secondary Outcomes (19)

• Normalization of LDH

  Through week 52

• Adequate control of hemolysis (LDH ≤1.5 × ULN)

  Through week 52

• Transfusion avoidance

  Through week 52

• Hemoglobin stabilization

  Through week 52

• Change in hemoglobin from baseline

  Through week 52

Study Arms (1)

Pozelimab + Cemdisiran Combo

EXPERIMENTAL

Drug: Pozelimab; Drug: Cemdisiran

Interventions

Pozelimab DRUG

Administered per the protocol

Also known as: REGN3918

Pozelimab + Cemdisiran Combo

Cemdisiran DRUG

Administered per the protocol

Also known as: ALN-CC5

Pozelimab + Cemdisiran Combo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of PNH confirmed by a history of high-sensitivity flow cytometry from prior testing
• Currently treated with marketed eculizumab, ravulizumab, or crovalimab at the labeled dose for at least 6 months
• 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
• At least 2 screening LDH values from different visits as described in the protocol
• 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

You may not qualify if:

• Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplants
• Body weight \<40 kilograms at screening visit
• Patients with a known or suspected C5 mutation that is refractory to their current C5i treatment as described in the protocol
• Any active or ongoing infection within 2 weeks of screening or during the screening period or any recent infection as described in the protocol
• Known hereditary complement deficiency

Sponsors & Collaborators

• Regeneron Pharmaceuticals: lead

Study Sites (19)

Hospital das Clinicas da FMRP USP

Ribeirão Preto, São Paulo, 14051-260, Brazil

RECRUITING

Faculdade de Medicina do ABC

Santo André, São Paulo, 09060 870, Brazil

RECRUITING

CISSS-CA

Lévis, Quebec, G6V 3Z1, Canada

RECRUITING

Universita Cattolica del Sacro Cuore - Policlinico Universitario A. Gemelli

Rome, Lazio, 00168, Italy

RECRUITING

AOU Careggi

Florence, Tuscany, 50134, Italy

RECRUITING

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

Torino, 10126, Italy

RECRUITING

In-Vivo Sp. z o.o.

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

RECRUITING

Seoul National University Hospital

Seoul, 03080, South Korea

RECRUITING

Severance Hospital

Seoul, 03722, South Korea

RECRUITING

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

Seoul, 065791, South Korea

RECRUITING

Ewha Womans University Mokdong Hospital

Seoul, 07985, South Korea

RECRUITING

Samsung Medical Center

Seoul, 6351, South Korea

RECRUITING

Hospital Universitario Basurto

Bilbao, Vizcaya, 48013, Spain

RECRUITING

Institut Catala d'Oncologia

Barcelona, 08908, Spain

RECRUITING

Hospital San Pedro de Alcantara

Cáceres, 10003, Spain

RECRUITING

Hospital General JM Morales Meseguer

Murcia, 30008, Spain

RECRUITING

Hospital Universitario de Salamanca

Salamanca, 37007, Spain

RECRUITING

Istanbul University Istanbul Faculty of Medicine

Istanbul, 34418, Turkey (Türkiye)

RECRUITING

Ege University

Izmir, 35100, Turkey (Türkiye)

RECRUITING

MeSH Terms

Conditions

Hemoglobinuria, Paroxysmal

Condition Hierarchy (Ancestors)

Anemia, Hemolytic; Anemia; Hematologic Diseases; Hemic and Lymphatic Diseases; Myelodysplastic Syndromes; Bone Marrow Diseases

Study Officials

• Clinical Trial Management

  Regeneron Pharmaceuticals

  STUDY DIRECTOR

Central Study Contacts

Clinical Trials Administrator

CONTACT

844-734-6643; clinicaltrials@regeneron.com

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 13, 2025
• First Posted: September 4, 2025
• Study Start: June 11, 2026
• Primary Completion (Estimated): January 18, 2030
• Study Completion (Estimated): December 4, 2031
• Last Updated: July 9, 2026

Record last verified: 2026-07

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR, ANALYTIC CODE
• Time Frame: When Regeneron has: * received marketing authorization from major health authorities (e.g., FDA, European Medicines Agency (EMA), Pharmaceuticals and Medical Devices Agency (PMDA), etc.) for the product and indication or has globally discontinued development of the product for all indications on or after April 2020 and has no plans for future development * made the study results publicly available (e.g., scientific publication, scientific conference, clinical trial registry) * the legal authority to share the data, and * ensured the ability to protect participant privacy
• Access Criteria: Qualified researchers can submit a proposal for access to individual patient or aggregate level data from a Regeneron-sponsored clinical trial through Vivli. Regeneron's Independent Research Request Evaluation Criteria can be found at: https://www.regeneron.com/sites/default/files/Regeneron-External-Data-Sharing-Policy-and-Independent-Research-Request-Evaluation-Criteria.pdf

Locations

CA (1); KR (5); IT (3); ES (5); PL (1); TU (2); BR (2)