Phase
Condition
Aplastic Anemia
White Cell Disorders
Anemia
Treatment
Cemdisiran
Pozelimab
Clinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Patients Entering from the Parent Study
Patients with PNH who have completed, without permanent discontinuation, studytreatment in the parent study (R3918-PNH-2021[NCT05133531]), including thepost-Open-label treatment period (OLTP) transition period, if applicable.
Willing and able to comply with clinic visits and study-related procedures,including meningococcal vaccinations required per protocol.
Patients Entering with C5 polymorphism
Patients with PNH who have a documented C5 polymorphism rendering them refractory toeculizumab or ravulizumab (eg, p.Arg885His, p.Arg885Cys), as described in theprotocol
Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNHgranulocytes or monocytes
Active disease, as defined by the presence of 1 or more PNH-related sign or symptomas described in the protocol
LDH level ≥2 × upper limit of normal (ULN) at the screening visit
Willing and able to comply with clinic visits and study-related procedures,including meningococcal vaccinations required per protocol
Exclusion
Key Exclusion Criteria:
Patients Entering from the Parent Study
Significant protocol deviation(s) in the parent study based on the investigator'sjudgment and to the extent that these would (if continued) impact the studyobjectives and/or safety of the patient
Any new condition or worsening of an existing condition which, in the opinion of theinvestigator, would make the patient unsuitable for enrollment or could interferewith the patient participating in or completing the study
Patients Entering with C5 polymorphism
Prior treatment with complement inhibitors within 5 half-lives of the respectiveagent prior to screening, except for prior eculizumab or ravulizumab which are notexclusionary
Receipt of an organ transplant, history of bone marrow transplantation or otherhematologic transplant
Not meeting meningococcal vaccination requirements and, at a minimum, documentationof quadrivalent meningococcal vaccination within 5 years prior to enrollment andserotype B vaccine within 3 years prior to enrollment as described in the protocol
Positive hepatitis B surface antigen or hepatitis C virus Ribonucleic acid (RNA)during screening
Patients with known HIV with history of opportunistic infections in the last 1 yearas described in the protocol
Known hereditary complement deficiency
Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
Documented history of liver cirrhosis or patients with liver disease with evidenceof current impaired liver function or patients with elevations in Alanineaminotransferase (ALT) or Aspartate aminotransferase (AST) (unrelated to PNH or itscomplications) as described in the protocol
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Study Design
Connect with a study center
Toronto General Hospital
Toronto, Ontario M5G2 C4
CanadaActive - Recruiting
Semmelweis University/Semmelweis Egyetem
Budapest, 1083
HungaryActive - Recruiting
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi, Delhi 110085
IndiaActive - Recruiting
Bhagwan Mahaveer Cancer Hospital & Research Centre
Jaipur, 302017
IndiaActive - Recruiting
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur, 302017
IndiaActive - Recruiting
Aou Careggi
Firenze, Forence 50139
ItalyActive - Recruiting
Azienda Ospedaliera Careggi
Firenze, Forence 50139
ItalyActive - Recruiting
Azienda Ospedaliera Città della Salute e della Scienza di Torino
Firenze, 50139
ItalyActive - Recruiting
SC Hematology, AOU Città della Salute e della Scienza di Torino
Torino, 10126
ItalyActive - Recruiting
University of Tsukuba Hospital
Tsukuba, Ibaraki 305-8576
JapanActive - Recruiting
St. Vincent Hospital
Suwon, Gyeonggi 16247
Korea, Republic ofActive - Recruiting
Ajou University Medical Center
Suwon-si, Gyeonggi-do 16499
Korea, Republic ofActive - Recruiting
St. Vincent Hospital
Suwon-si, Gyeonggi-do 16247
Korea, Republic ofActive - Recruiting
Gachon University Gil Medical Center
Incheon, Namdong-Gu 405-760
Korea, Republic ofActive - Recruiting
Pusan National University Hospital
Busan, 49241
Korea, Republic ofActive - Recruiting
Ewha Womans University Mokdong Hospital
Seoul, 07985
Korea, Republic ofActive - Recruiting
Samsung Medical Center
Seoul, 06351
Korea, Republic ofActive - Recruiting
Severance Hospital Yonsei University Health System
Seoul, 03722
Korea, Republic ofActive - Recruiting
Ajou University Medical Center
Suwon, 16499
Korea, Republic ofActive - Recruiting
Hospital Tg Ampuan Afzan
Kuantan, Pahang 25200
MalaysiaActive - Recruiting
Hospital Queen Elizabeth
Kota Kinabalu, Sabah 88200
MalaysiaActive - Recruiting
Hospital Ampang
Ampang, Selangor 68000
MalaysiaActive - Recruiting
St Lukes Medical Center
Quezon City, National Capital Region 1634
PhilippinesActive - Recruiting
Szpital Uniwersytecki Nr2 Bydgoszcz
Bydgoszcz, 85-168
PolandActive - Recruiting
Prof Dr Ion Chiricuta Cancer Institute
Cluj Napoca, Cluj 400015
RomaniaActive - Recruiting
Prof Dr Ion Chiricuta Institute of Oncology
Cluj-Napoca, 400015
RomaniaSite Not Available
National University Hospital
Singapore, 119074
SingaporeActive - Recruiting
Hospital Universitario Basurto
Bilbao, Bizkaia 48013
SpainActive - Recruiting
Hospital Clinic de Barcelona
Barcelona, 08036
SpainActive - Recruiting
Hospital Universitario de Basurto
Bilbao, 48013
SpainActive - Recruiting
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan, Hunan Province 33305
TaiwanActive - Recruiting
Hualien Tzu Chi Hospital
Hualien City, 97002
TaiwanActive - Recruiting
China Medical University Hospital
Taichung, 40447
TaiwanActive - Recruiting
National Taiwan University Hospital
Taipei, 10002
TaiwanActive - Recruiting
Tri-Service General Hospital
Taipei City, 114
TaiwanActive - Recruiting
Chang Gung Memorial Hospital- Linkou Branch
Taoyuan, 333
TaiwanActive - Recruiting
Prince Of Songkla Hospital, Prince Of Songkhla University
Hat-Yai, Songkhla 90110
ThailandActive - Recruiting
King Chulalongkorn Memorial Hospital
Bangkok, 10330
ThailandActive - Recruiting
Chiang Mai University
Chiang Mai, 50200
ThailandActive - Recruiting
Songklanagarind Hospital
Hat Yai, 90110
ThailandSite Not Available
Srinagarind Hospital
Khon Kaen, 40002
ThailandActive - Recruiting
Maharaj Nakorn Chiang Mai Hospital
Muang, 50200
ThailandSite Not Available
Ege University Faculty of Medicine
Bornova, Izmir 35100
TurkeyActive - Recruiting
Ege Universitesi Tip Fakultesi Hastanesi
Izmir, 35100
TurkeySite Not Available
Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology
Leeds, LS97TF
United KingdomActive - Recruiting
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