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Clinical Trials in the UK / NCT05744921
Recruiting Phase 3

A Study in Adult Patients With Paroxysmal Nocturnal Hemoglobinuria (PNH) to Evaluate How Safe Long-term Treatment With Pozelimab + Cemdisiran Combination Therapy is and How Well it Works

NCT05744921 · tracked via the Priya Life Science UK tracker
Sponsor
Regeneron Pharmaceuticals
Phase
Phase 3
Started
2023-03-07
Last updated
2026-09-17

Condition(s) studied

Paroxysmal Nocturnal Hemoglobinuria

Investigational drug(s) / intervention(s)

PozelimabCemdisiran

Pozelimab: Administered per the protocol

Cemdisiran: Administered per the protocol

Study summary

This study is researching an experimental treatment combination with two experimental drugs called pozelimab and cemdisiran. The study is focused on people with paroxysmal nocturnal hemoglobinuria (PNH). The aim of this study is to see how safe and effective the pozelimab + cemdisiran combination is for people with PNH in the long term. The pozelimab + cemdisiran combination may be referred to as "study drugs" in this section.

This study is looking at several other research questions, including:

* How effective is the pozelimab + cemdisiran combination?
* What side effects may happen from taking the study drugs?
* How much of each study drug is in the blood at different times?
* Whether the body makes antibodies against the study drugs (which could make the drugs less effective or could lead to side effects)

Eligibility

Sex
ALL
Min age
18 Years
Max age
Healthy volunteers
No
Key Inclusion Criteria: Patients Entering from the Parent Study 1. Patients with PNH who have completed, without permanent discontinuation, study treatment in the parent study (R3918-PNH-2021\[NCT05133531\]), including the post-Open-label treatment period (OLTP) transition period, if applicable. 2. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol. Patients Entering with C5 polymorphism 1. Patients with PNH who have a documented C5 polymorphism rendering them refractory to eculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in the protocol 2. Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes 3. Active disease, as defined by the presence of 1 or more PNH-related sign or symptom as described in the protocol 4. LDH level ≥2 × upper limit of normal (ULN) at the screening visit 5. Willing and able to comply with clinic visits and study-related procedures, including meningococcal vaccinations required per protocol Key Exclusion Criteria: Patients Entering from the Parent Study 1. Significant protocol deviation(s) in the parent study based on the investigator's judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient 2. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study Patients Entering with C5 polymorphism 1. Prior treatment with complement inhibitors within 5 half-lives of the respective agent prior to screening, except for prior eculizumab or ravulizumab which are not exclusionary 2. Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant 3. Not meeting meningococcal vaccination requirements and, at a minimum, documentation of quadrivalent meningococcal vaccination within 5 years prior to enrollment and serotype B vaccine within 3 years prior to enrollment as described in the protocol 4. Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA) during screening 5. Patients with known HIV with history of opportunistic infections in the last 1 year as described in the protocol 6. Known hereditary complement deficiency 7. Documented history of active, uncontrolled, ongoing systemic autoimmune diseases 8. Documented history of liver cirrhosis or patients with liver disease with evidence of current impaired liver function or patients with elevations in Alanine aminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or its complications) as described in the protocol Note: Other protocol-defined Inclusion/ Exclusion Criteria apply

Primary outcome measure(s)

Trial sites (47)

FacilityCityRegionStatus
Toronto General Hospital Toronto Ontario Recruiting
Hospital Pablo Tobon Uribe Medellín Antioquia Recruiting
Semmelweis University/Semmelweis Egyetem Budapest Hungary Recruiting
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims Kochi Kerala Recruiting
K J Somaiya Super Specialty Hospital & Research Centre Mumbai Maharashtra Recruiting
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus New Delhi National Capital Territory of Delhi Recruiting
Postgraduate Institute of Medical Education & Research Chandigarh India Recruiting
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC) Jaipur India Recruiting
Aou Careggi Florence Firenze Recruiting
SC Hematology, AOU Città della Salute e della Scienza di Torino Torino Italy Recruiting
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital Nagoya Aichi-ken Withdrawn
University of Tsukuba Hospital Tsukuba Ibaraki Recruiting
Jordan University Hospital (JUH) Amman Jordan Recruiting
Hospital Tg Ampuan Afzan Kuantan Pahang Recruiting
Hospital Queen Elizabeth Kota Kinabalu Sabah Recruiting
Hospital Ampang Ampang Selangor Recruiting
Clinica San Felipe Lima Peru Recruiting
St Lukes Medical Center Quezon City National Capital Region Recruiting
In-Vivo Sp. z o.o. Bydgoszcz Kuyavian-Pomeranian Voivodeship Recruiting
University Clinical Center Medical University of Gdansk Gdansk Pomeranian Voivodeship Recruiting
Szpital Uniwersytecki Nr2 Bydgoszcz Bydgoszcz Poland Completed
Prof Dr Ion Chiricuta Cancer Institute Cluj-Napoca Cluj Recruiting
National University Hospital Singapore Singapore Recruiting
St. Vincent Hospital Suwon Gyeonggi-do Recruiting
Ajou University Medical Center Suwon Gyeonggi-do Recruiting
Gachon University Gil Medical Center Incheon Namdong-Gu Recruiting
Pusan National University Hospital Busan South Korea Recruiting
Severance Hospital Yonsei University Health System Seoul South Korea Recruiting
Samsung Medical Center Seoul South Korea Recruiting
The Catholic University of Korea, Seoul St. Mary's Hospital Seoul South Korea Recruiting
Ewha Womans University Mokdong Hospital Seoul South Korea Recruiting
Hospital Universitario Basurto Bilbao Vizcaya Recruiting
Hospital Clinic de Barcelona Barcelona Spain Recruiting
China Medical University Hospital Taichung Central Taiwan Recruiting
Chang Gung Memorial Hospital - Linkou Branch Taoyuan Hunan Province Recruiting
Changhua Christian Hospital Changhua Taiwan Recruiting
Hualien Tzu Chi Hospital Hualien City Taiwan Recruiting
Taichung Veterans General Hospital (VGHTC) Taichung Taiwan Recruiting
National Taiwan University Hospital Taipei Taiwan Recruiting
Tri-Service General Hospital Taipei Taiwan Recruiting

+ 7 more sites — see the full list on the official registry below.

More Regeneron Pharmaceuticals trials in the UK

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 NCT05744921 on ClinicalTrials.gov ↗ ← All trials in the UK