Phase
Condition
Aplastic Anemia
Anemia
Hemoglobinuria, Paroxysmal
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 M5G 2C4
CanadaActive - Recruiting
Toronto General Hospital
Toronto 6167865, Ontario 6093943 M5G 2C4
CanadaSite Not Available
Hospital Pablo Tobon Uribe
Medellín, Antioquia 050034
ColombiaActive - Recruiting
Hospital Pablo Tobon Uribe
Medellín 3674962, Antioquia 3689815 050034
ColombiaSite Not Available
Semmelweis University/Semmelweis Egyetem
Budapest, 1083
HungaryActive - Recruiting
Semmelweis University/Semmelweis Egyetem
Budapest 3054643, 1083
HungarySite Not Available
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
Kochi, Kerala 682041
IndiaActive - Recruiting
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
Kochi 1273874, Kerala 1267254 682041
IndiaSite Not Available
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai, Maharashtra 400022
IndiaActive - Recruiting
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai 1275339, Maharashtra 1264418 400022
IndiaSite Not Available
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi, National Capital Territory of Delhi 110085
IndiaActive - Recruiting
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi 1261481, National Capital Territory of Delhi 1273293 110085
IndiaSite Not Available
Bhagwan Mahaveer Cancer Hospital & Research Centre
Jaipur, 302017
IndiaActive - Recruiting
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur, 302017
IndiaActive - Recruiting
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur 1269515, 302017
IndiaSite Not Available
Aou Careggi
Florence, Firenze 50139
ItalyActive - Recruiting
Aou Careggi
Florence 3176959, Firenze 50139
ItalySite Not Available
Aou Careggi
Firenze, Florence 50139
ItalySite Not Available
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
SC Hematology, AOU Città della Salute e della Scienza di Torino
Torino 8980539, 10126
ItalySite Not Available
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aichi-ken 466-8650
JapanSite Not Available
University of Tsukuba Hospital
Tsukuba, Ibaraki 305-8576
JapanActive - Recruiting
University of Tsukuba Hospital
Tsukuba 2110681, Ibaraki 2112669 305-8576
JapanSite Not Available
Jordan University Hospital (JUH)
Amman, 11942
JordanActive - Recruiting
Jordan University Hospital (JUH)
Amman 250441, 11942
JordanSite Not Available
St. Vincent Hospital
Suwon, Gyeonggi 16247
Korea, Republic ofSite Not Available
Ajou University Medical Center
Suwon-si, Gyeonggi-do 16499
Korea, Republic ofActive - Recruiting
St. Vincent Hospital
Suwon-si, Gyeonggi-do 16247
Korea, Republic ofSite Not Available
Gachon University Gil Medical Center
Incheon, Namdong-Gu 405-760
Korea, Republic ofSite Not Available
Samsung Medical Center
Seoul, Seoul Teugbyeolsi 06351
Korea, Republic ofSite Not Available
Pusan National University Hospital
Busan, 49241
Korea, Republic ofSite Not Available
Ewha Womans University Mokdong Hospital
Seoul, 07985
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 Tg Ampuan Afzan
Kuantan 1735227, Pahang 1733042 25200
MalaysiaSite Not Available
Hospital Queen Elizabeth
Kota Kinabalu, Sabah 88200
MalaysiaActive - Recruiting
Hospital Queen Elizabeth
Kota Kinabalu 1733432, Sabah 1733039 88200
MalaysiaSite Not Available
Hospital Ampang
Ampang, Selangor 68000
MalaysiaActive - Recruiting
Clinica San Felipe
Lima, 15072
PeruActive - Recruiting
Clinica San Felipe
Lima 3936456, 15072
PeruSite Not Available
St Lukes Medical Center
Quezon City, National Capital Region 1634
PhilippinesActive - Recruiting
St Lukes Medical Center
Quezon City 1692192, National Capital Region 7521311 1634
PhilippinesSite Not Available
In-Vivo Sp. z o.o.
Bydgoszcz, Kuyavian-Pomeranian Voivodeship 85-048
PolandActive - Recruiting
IN-VIVO Sp. z o.o.
Bydgoszcz 3102014, Kuyavian-Pomeranian Voivodeship 3337500 85-048
PolandSite Not Available
University Clinical Center Medical University of Gdansk
Gdansk, Pomeranian Voivodeship 80-214
PolandActive - Recruiting
University Clinical Center Medical University of Gdansk
Gdansk 3099434, Pomeranian Voivodeship 3337496 80-214
PolandSite Not Available
Szpital Uniwersytecki Nr2 Bydgoszcz
Bydgoszcz, 85-168
PolandActive - Recruiting
Szpital Uniwersytecki Nr2 Bydgoszcz
Bydgoszcz 3102014, 85-168
PolandActive - Recruiting
Prof Dr Ion Chiricuta Cancer Institute
Cluj Napoca, Cluj 400015
RomaniaSite Not Available
Prof Dr Ion Chiricuta Cancer Institute
Cluj-Napoca, Cluj 400015
RomaniaActive - Recruiting
Prof Dr Ion Chiricuta Cancer Institute
Cluj-Napoca 681290, Cluj 681291 400015
RomaniaSite Not Available
Prof Dr Ion Chiricuta Institute of Oncology
Cluj-Napoca, 400015
RomaniaSite Not Available
National University Hospital
Singapore, 119074
SingaporeActive - Recruiting
National University Hospital
Singapore 1880252, 119074
SingaporeSite Not Available
Ajou University Medical Center
Suwon, Gyeonggi-do 16499
South KoreaActive - Recruiting
St. Vincent Hospital
Suwon, Gyeonggi-do 16247
South KoreaActive - Recruiting
Ajou University Medical Center
Suwon 1835553, Gyeonggi-do 1841610 16499
South KoreaActive - Recruiting
St. Vincent Hospital
Suwon 1835553, Gyeonggi-do 1841610 16247
South KoreaSite Not Available
Gachon University Gil Medical Center
Incheon, Namdong-Gu 21565
South KoreaActive - Recruiting
Gachon University Gil Medical Center
Incheon 1843564, Namdong-Gu 21565
South KoreaSite Not Available
Pusan National University Hospital
Busan, 49241
South KoreaActive - Recruiting
Pusan National University Hospital
Busan 1838524, 49241
South KoreaSite Not Available
Ewha Womans University Mokdong Hospital
Seoul, 07985
South KoreaActive - Recruiting
Samsung Medical Center
Seoul, 06351
South KoreaActive - Recruiting
Severance Hospital Yonsei University Health System
Seoul, 03722
South KoreaActive - Recruiting
Ewha Womans University Mokdong Hospital
Seoul 1835848, 07985
South KoreaSite Not Available
Samsung Medical Center
Seoul 1835848, 06351
South KoreaActive - Recruiting
Severance Hospital Yonsei University Health System
Seoul 1835848, 03722
South KoreaActive - Recruiting
Hospital Universitario Basurto
Bilbao, Vizcaya 48013
SpainActive - Recruiting
Hospital Universitario Basurto
Bilbao 3128026, Vizcaya 48013
SpainSite Not Available
Hospital Clinic de Barcelona
Barcelona, 08036
SpainActive - Recruiting
Hospital Clinic de Barcelona
Barcelona 3128760, 08036
SpainSite Not Available
Hospital Universitario de Basurto
Bilbao, 48013
SpainActive - Recruiting
China Medical University Hospital
Taichung, Central Taiwan 40447
TaiwanActive - Recruiting
China Medical University Hospital
Taichung 1668399, Central Taiwan 40447
TaiwanSite Not Available
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan, Hunan Province 33305
TaiwanSite Not Available
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan District, Hunan Province 33305
TaiwanActive - Recruiting
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan District 1667905, Hunan Province 33305
TaiwanSite Not Available
Hualien Tzu Chi Hospital
Hualien City, 97002
TaiwanActive - Recruiting
Hualien Tzu Chi Hospital
Hualien City 1674504, 97002
TaiwanSite Not Available
National Taiwan University Hospital
Taipei, 10002
TaiwanActive - Recruiting
Tri-Service General Hospital
Taipei, 11490
TaiwanActive - Recruiting
National Taiwan University Hospital
Taipei 1668341, 10002
TaiwanActive - Recruiting
Tri-Service General Hospital
Taipei 1668341, 11490
TaiwanSite Not Available
Tri-Service General Hospital
Taipei City, 114
TaiwanSite Not Available
Chang Gung Memorial Hospital- Linkou Branch
Taoyuan, 333
TaiwanActive - Recruiting
Prince Of Songkla Hospital, Prince Of Songkhla University
Hat Yai, Changwat Songkhla 90110
ThailandActive - Recruiting
Prince Of Songkla Hospital, Prince Of Songkhla University
Hat Yai 1610780, Changwat Songkhla 1606146 90110
ThailandSite Not Available
Prince Of Songkla Hospital, Prince Of Songkhla University
Hat-Yai, Songkhla 90110
ThailandSite Not Available
King Chulalongkorn Memorial Hospital
Bangkok, 10330
ThailandActive - Recruiting
King Chulalongkorn Memorial Hospital
Bangkok 1609350, 10330
ThailandSite Not Available
Chiang Mai University
Chiang Mai, 50200
ThailandActive - Recruiting
Chiang Mai University
Chiang Mai 1153671, 50200
ThailandSite Not Available
Songklanagarind Hospital
Hat Yai, 90110
ThailandSite Not Available
Faculty of Medicine Khon Kaen University
Khon Kaen, 40002
ThailandActive - Recruiting
Srinagarind Hospital
Khon Kaen, 40002
ThailandSite Not Available
Faculty of Medicine Khon Kaen University
Khon Kaen 1609776, 40002
ThailandSite Not Available
Srinagarind Hospital
Khon Kaen 1609776, 40002
ThailandActive - Recruiting
Maharaj Nakorn Chiang Mai Hospital
Muang, 50200
ThailandSite Not Available
Ege University Faculty of Medicine
Bornova, Izmir 35100
TurkeySite Not Available
Ege Universitesi Tip Fakultesi Hastanesi
Izmir, 35100
TurkeySite Not Available
Ege University Faculty of Medicine
Bornova, İzmir 35100
Turkey (Türkiye)Active - Recruiting
Ege University Faculty of Medicine
Bornova 320857, İzmir 311044 35100
Turkey (Türkiye)Site Not Available
Istanbul University
Istanbul, 34418
Turkey (Türkiye)Active - Recruiting
Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology
Leeds, LS97TF
United KingdomActive - Recruiting
Leeds Teaching Hospitals NHS Trust - St. James Institute of Oncology
Leeds 2644688, LS97TF
United KingdomSite Not Available

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