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Clinical Trials in Turkey / NCT07154745
Recruiting Phase 3

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 · tracked via the Priya Life Science Turkey tracker
Phase
Phase 3
Started
2026-06-11
Last updated
2026-08-05

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Investigational drug(s) / intervention(s)

Pozelimab →Cemdisiran →

Pozelimab: Administered per the protocol

Cemdisiran: Administered per the protocol

Study 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)

Eligibility

Sex
ALL
Min age
18 Years
Max age
—
Healthy volunteers
No
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

Primary outcome measure(s)

Trial sites (20)

FacilityCityRegionStatus
Hospital das Clinicas da FMRP USP Ribeirão Preto São Paulo Recruiting
Faculdade de Medicina do ABC Santo André São Paulo Recruiting
Hospital Santa Marcelina São Paulo Brazil Recruiting
CISSS-CA Lévis Quebec Recruiting
Universita Cattolica del Sacro Cuore - Policlinico Universitario A. Gemelli Rome Lazio Recruiting
AOU Careggi Florence Tuscany Recruiting
SC Hematology, AOU Città della Salute e della Scienza di Torino Torino Italy Recruiting
In-Vivo Sp. z o.o. Bydgoszcz Kuyavian-Pomeranian Voivodeship Recruiting
Seoul National University Hospital Seoul South Korea Recruiting
Severance Hospital Seoul South Korea Recruiting
Seoul St. Mary's Hospital - The Catholic University of Korea Seoul South Korea Recruiting
Ewha Womans University Mokdong Hospital Seoul South Korea Recruiting
Samsung Medical Center Seoul South Korea Recruiting
Hospital Universitario Basurto Bilbao Vizcaya Recruiting
Institut Catala d'Oncologia Barcelona Spain Recruiting
Hospital San Pedro de Alcantara Cáceres Spain Recruiting
Hospital General JM Morales Meseguer Murcia Spain Recruiting
Hospital Universitario de Salamanca Salamanca Spain Recruiting
Istanbul University Istanbul Faculty of Medicine Istanbul Turkey (Türkiye) Recruiting
Ege University Izmir Turkey (Türkiye) Recruiting

More Regeneron Pharmaceuticals trials in Turkey

Other trials for the same condition

Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07154745 on ClinicalTrials.gov ↗ ← All trials in Turkey