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
Condition(s) studied
Investigational drug(s) / intervention(s)
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
Primary outcome measure(s)
- Incidence of treatment-emergent serious adverse events (SAEs) — Up to week 108
An SAE is any untoward medical occurrence that at any dose: * Results in death * Is life-threatening * Requires in-patient hospitalization or prolongation of existing hospitalization. * Results in persistent or significant disability/incapacity * Is a congenital anomaly/birth defect. * Is an important medical event - Severity of treatment-emergent SAEs — Up to week 108
- Incidence of treatment emergent adverse events of special interest (AESIs) — Up to week 108
An AESI (serious or non-serious) is one of scientific and medical concern specific to the sponsor's product or program, for which ongoing monitoring and rapid communication by the Investigator to the sponsor can be appropriate. Such an event might warrant further investigation in order to characterize and understand it - Severity of treatment emergent AESIs — Up to week 108
- Incidence of adverse events (AEs) leading to permanent treatment discontinuation — Up to week 108
Any untoward medical occurrence in a patient administered a study drug which may or may not have a causal relationship with the study drug. - Severity of adverse events (AEs) leading to permanent treatment discontinuation — Up to week 108
Any untoward medical occurrence in a patient administered a study drug which may or may not have a causal relationship with the study drug. - Percent change from baseline in lactate dehydrogenase (LDH) — Baseline to week 36
Trial sites (48)
| Facility | City | Region | Status |
|---|---|---|---|
| A Beneficencia Portuguesa de Sao Paulo, BP Mirante | São Paulo | Brazil | Recruiting |
| 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 |
+ 8 more sites — see the full list on the official registry below.
More Regeneron Pharmaceuticals trials in Japan
Other trials for the same condition
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 Japan