Phase
Condition
White Cell Disorders
Hemoglobinuria, Paroxysmal
Aplastic Anemia
Treatment
Cemdisiran
Pozelimab
Eculizumab
Clinical Study ID
Ages > 18 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNHgranulocytes or monocytes as described in the protocol
Active disease, as defined by the presence of 1 or more PNH-related signs orsymptoms as described in the protocol
LDH level ≥2 × ULN at the screening visit
Willing and able to comply with clinic/remote visits and study-related procedures,including completion of the full series of meningococcal vaccinations required perprotocol
Exclusion
Key Exclusion Criteria:
Prior treatment with eculizumab within 3 months prior to screening, ravulizumabwithin 6 months prior to screening, or other complement inhibitors within 5half-lives of the respective agent prior to screening
Receipt of an organ transplant, history of bone marrow transplantation or otherhematologic transplant
Body weight <40 kilograms at screening visit
Planned use of any complement inhibitor therapy other than study drugs during thetreatment period
Not meeting meningococcal vaccination requirements and, at a minimum documentationof quadrivalent meningococcal vaccination within 5 years prior to the screeningvisit and serotype B vaccine within 3 years prior to the screening visit asdescribed in the protocol.
Any contraindication for receiving Neisseria meningitidis vaccinations (serotypesACWY and B).
Unable to take antibiotics for meningococcal prophylaxis (if required by localravulizumab [Cohort A] or eculizumab [Cohort B] prescribing information, whereavailable, or national guidelines/local practice, or if necessary whenadministration of the first dose of the quadrivalent meningococcal vaccine [serotypeACWY] or the second dose of the serotype B meningococcal vaccine [when available] isless than 2 weeks prior to study treatment initiation) as described in the protocol
Any active, ongoing infection or a recent infection requiring ongoing systemictreatment with antibiotics, antivirals, or antifungals within 2 weeks of screeningor during the screening period
Documented history of active, uncontrolled, ongoing systemic autoimmune diseases
Note: Other protocol-defined Inclusion/ Exclusion Criteria apply
Study Design
Connect with a study center
Centro de Estudos e Pesquisas em Hematologia e Oncologia
Santo André 3449701, São Paulo 3448433 09060-650
BrazilActive - Recruiting
A Beneficencia Portuguesa de Sao Paulo, BP Mirante
São Paulo 3448439, 01321-001
BrazilActive - Recruiting
Casa de Saude Santa Marcelina
São Paulo 3448439, 08270-070
BrazilActive - Recruiting
Toronto General Hospital
Toronto, Ontario M5G2 C4
CanadaSite Not Available
Toronto General Hospital
Toronto 6167865, Ontario 6093943 M5G 2C4
CanadaActive - Recruiting
Peking Union Medical College Hospital
Beijing, Beijing 100032
ChinaSite Not Available
Peking Union Medical College Hospital
Beijing 1816670, Beijing Municipality 2038349 100032
ChinaCompleted
Hospital Pablo Tobon Uribe
Medellin, Antioquia 050034
ColombiaSite Not Available
Hospital Pablo Tobon Uribe
Medellín 3674962, Antioquia 3689815 050034
ColombiaActive - Recruiting
George Papanikolaou Hospital
Thessaloniki, 57010
GreeceSite Not Available
George Papanikolaou Hospital
Thessaloniki 734077, 57010
GreeceActive - Recruiting
Semmelweis Egyetem
Budapest, 1089
HungaryActive - Recruiting
Semmelweis University
Budapest, 1088
HungarySite Not Available
Semmelweis University
Budapest 3054643, 1083
HungaryActive - Recruiting
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi, Delhi 110085
IndiaSite Not Available
Rajiv Gandhi Cancer Institute and Research Centre
New Delhi, Delhi 110085
IndiaActive - Recruiting
Malabar Cancer Center, Kerala
Kannur, Kerala 670103
IndiaSite Not Available
Malabar Cancer Centre
Kannur, Kerala 670103
IndiaSite Not Available
Amrita Institute of Medical Sciences (AIMS) and Research Centre
Kochi, Kerala 682041
IndiaActive - Recruiting
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
Kochi, Kerala 682041
IndiaSite Not Available
Malabar Cancer Center, Kerala
Kannur 1274987, Kerala 1267254 670103
IndiaActive - Recruiting
Amrita Institute of Medical Sciences (AIMS) and Research Centre Aims
Kochi 1273874, Kerala 1267254 682041
IndiaActive - Recruiting
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai, Maharashtra 400022
IndiaSite Not Available
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai 1275339, Maharashtra 1264418 400022
IndiaActive - Recruiting
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi 1261481, National Capital Territory of Delhi 1273293 110085
IndiaActive - Recruiting
Postgraduate Institute of Medical Education & Research (PGIMER)
Chandigarh, Punjab 1259223 708248
IndiaActive - Recruiting
Bhagwan Mahaveer Cancer Hospital & Research Centre
Jaipur, Rajasthan 302017
IndiaActive - Recruiting
King George Hospital
Lucknow, Uttar Pradesh 226003
IndiaSite Not Available
Kings George's Medical University
Lucknow, Uttar Pradesh 226014
IndiaSite Not Available
King George Hospital
Lucknow 1264733, Uttar Pradesh 1253626 226003
IndiaActive - Recruiting
Sanjay Gandhi Postgraduate Institute of Medical Sciences
Lucknow 1264733, Uttar Pradesh 1253626 226014
IndiaActive - Recruiting
Postgraduate Institute of Medical Education and Research
Chandigarh, 160012
IndiaActive - Recruiting
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur, 302017
IndiaSite Not Available
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur 1269515, 302017
IndiaActive - Recruiting
Malabar Cancer Center, Kerala
Kerala, 670103
IndiaSite Not Available
King George Hospital
Uttar Pradesh, 226003
IndiaSite Not Available
Aou Careggi
Florence 3176959, Firenze 50139
ItalyActive - Recruiting
Aou Careggi
Firenze, Florence 50139
ItalySite Not Available
Fondazione Policlinico Universitario A Gemelli
Roma, Lazio 168
ItalySite Not Available
Azienda Ospedaliera Città della Salute e della Scienza di Torino
Torino, Piemonte 10126
ItalySite Not Available
Fondazione Policlinico Universitario A. Gemelli - IRCCS
Rome 3169070, Roma 00168
ItalyActive - Recruiting
Fondazione Policlinico Universitrio a. Gemelli - IRCCS
Rome 3169070, Roma 00168
ItalyActive - Recruiting
Azienda Ospedaliera Universitaria Careggi
Firenze, 50134
ItalyActive - Recruiting
Fondazione Policlinico Universitrio a. Gemelli - IRCCS
Rome, 000168
ItalySite Not Available
Hematology Citta della Salute e della Scienza di Torino
Turin, 10126
ItalySite Not Available
Hematology Citta della Salute e della Scienza di Torino
Turin 3165524, 10126
ItalyActive - Recruiting
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
Nagoya 1856057, Aichi-ken 1865694 466-8650
JapanActive - Recruiting
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aiti 466-8650
JapanSite Not Available
Ogaki Municipal Hospital
Ogaki, Gifu 503-8502
JapanSite Not Available
Ogaki Municipal Hospital
Ogaki city, Gifu 503-8502
JapanSite Not Available
Ogaki Municipal Hospital
Ōgaki 1854703, Gifu 1863640 503-8502
JapanActive - Recruiting
University of Tsukuba Hospital
Tsukuba, Ibaraki 305-8576
JapanSite Not Available
University of Tsukuba Hospital
Tsukuba-shi, Ibaraki 305-8576
JapanSite Not Available
University of Tsukuba Hospital
Tsukuba 2110681, Ibaraki 2112669 305-8576
JapanActive - Recruiting
Matsushita Memorial Hospital
Moriguchi, Osaka 570-8540
JapanSite Not Available
Panasonic Health Insurance Organization Matsushita Memorial Hospital
Moriguchi-city, Osaka 570-8540
JapanSite Not Available
Matsushita Memorial Hospital
Moriguchi 1856584, Osaka 1853904 570-8540
JapanActive - Recruiting
NTT Medical Center Tokyo
Shinagawa-ku, Tokyo 1850144 141-8625
JapanActive - Recruiting
Jordan University Hospital (JUH)
Amman, 11942
JordanSite Not Available
University of Jordan
Amman, 11196
JordanActive - Recruiting
Jordan University Hospital (JUH)
Amman 250441, 11942
JordanActive - Recruiting
Ajou University Medical Center
Suwon, Gyeonggi 16499
Korea, Republic ofSite Not Available
St. Vincent Hospital
Suwon-si, Gyeonggi-do 16247
Korea, Republic ofSite Not Available
Gachon University Gil Medical Center
Incheon, Namdong-Gu 21565
Korea, Republic ofSite Not Available
Pusan National University Hospital
Busan, 49241
Korea, Republic ofSite Not Available
Ajou University Medical Center
Gyeonggi-do, 16499
Korea, Republic ofSite Not Available
Ajou University Medical Center
Gyeonggido, 16499
Korea, Republic ofSite Not Available
Ewha Womans University Mokdong Hospital
Seoul, 07985
Korea, Republic ofActive - Recruiting
Korea University Hospital
Seoul, 02841
Korea, Republic ofActive - Recruiting
Samsung Medical Center
Seoul, 06351
Korea, Republic ofSite Not Available
Samsung Medical Center - PPDS
Seoul, 06351
Korea, Republic ofActive - Recruiting
Severance Hospital
Seoul, 03722
Korea, Republic ofActive - Recruiting
St. Vincent Hospital
Suwon, 16247
Korea, Republic ofSite Not Available
Hospital Tengku Ampuan Afzan
Kuantan, Pahang 25200
MalaysiaActive - Recruiting
Hospital Tg Ampuan Afzan
Kuantan, Pahang 25200
MalaysiaSite Not Available
Hospital Tg Ampuan Afzan
Kuantan 1735227, Pahang 1733042 25200
MalaysiaActive - Recruiting
Hospital Queen Elizabeth
Kota Kinabalu, Sabah 88586
MalaysiaSite Not Available
Hospital Queen Elizabeth
Kota Kinabalu 1733432, Sabah 1733039 88586
MalaysiaActive - Recruiting
Hospital Ampang
Ampang, Selangor 1733037 68000
MalaysiaActive - Recruiting
Queen Elizabeth Hospital - Kota Kinabalu
Kota Kinabalu, 88586
MalaysiaActive - Recruiting
Servicio de Hematologia del Hospital Universitario de la Uanl
Monterrey, Nuevo Leon 64460
MexicoSite Not Available
Hospital Universitario Dr. Jose Eleuterio González
Monterrey, Nuevo León 64460
MexicoActive - Recruiting
Servicio de Hematologia del Hospital Universitario de la Uanl
Monterrey 3995465, Nuevo León 3522542 64460
MexicoActive - Recruiting
Clinica San Felipe
Lima, 15072
PeruSite Not Available
Clinica San Felipe
Lima 3936456, 15072
PeruActive - Recruiting
St Lukes Medical Center
Quezon City, Central Luzon 1011
PhilippinesSite Not Available
St Lukes Medical Center
Quezon 1692199, Central Luzon 7521305 1011
PhilippinesActive - Recruiting
University Clinical Center Medical University of Gdansk
Gdansk, Pomeranian Voivodeship 80-214
PolandSite Not Available
University Clinical Center Medical University of Gdansk
Gdansk 3099434, Pomeranian Voivodeship 3337496 80-214
PolandActive - Recruiting
Klinika Hematologii, Szpital Uniwersytecki Nr 2 im. Jana Biziela w Bydgoszczy
Bydgoszcz, 85-168
PolandActive - Recruiting
Szpital Uniwersytecki Nr2 Bydgoszcz
Bydgoszcz, 85-168
PolandSite Not Available
Szpital Uniwersytecki Nr2 Bydgoszcz
Bydgoszcz 3102014, 85-168
PolandActive - Recruiting
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-214
PolandActive - Recruiting
Institute of Hematology and Transfusion Medicine
Warsaw, 02-776
PolandSite Not Available
Institute of Hematology and Transfusion Medicine
Warsaw 756135, 02-776
PolandActive - Recruiting
Instytut Hematologii i Transfuzjologii
Warszawa, 02-776
PolandSite Not Available
Ion Chiricuta Oncology Institute
Cluj-Napoca, Cluj 400015
RomaniaSite Not Available
Prof Dr I Chiricuta Institute of Oncology
Cluj-Napoca, Cluj 400015
RomaniaActive - Recruiting
Ion Chiricuta Oncology Institute
Cluj-Napoca 681290, Cluj 681291 400015
RomaniaActive - Recruiting
Filantropia Municipal Clinical Hospital
Craiova, Dolj 200143
RomaniaActive - Recruiting
Municipal Hospital Filantropia
Craiova, Dolj 200143
RomaniaSite Not Available
Municipal Hospital Filantropia
Craiova 680332, Dolj 679134 200143
RomaniaActive - Recruiting
Targu Mures Clinical County Emergency Hospital
Targu Mures, Mures 540136
RomaniaSite Not Available
Targu-Mures Emergency Clinical County Hospital
Targu Mures, Mures 540136
RomaniaActive - Recruiting
Targu Mures Clinical County Emergency Hospital
Târgu Mureş 665004, Mureș County 672628 540136
RomaniaActive - Recruiting
National University Hospital
Singapore, 119074
SingaporeSite Not Available
National University Hospital
Singapore 1880252, 119074
SingaporeActive - Recruiting
Ajou University Medical Center
Suwon 1835553, Gyeonggi-do 1841610 16499
South KoreaActive - Recruiting
St. Vincent Hospital
Suwon 1835553, Gyeonggi-do 1841610 16247
South KoreaActive - Recruiting
Gachon University Gil Medical Center
Incheon 1843564, Namdong-Gu 21565
South KoreaActive - Recruiting
Pusan National University Hospital
Busan 1838524, 49241
South KoreaActive - Recruiting
Ewha Womans University Mokdong Hospital
Seoul 1835848, 07985
South KoreaActive - Recruiting
Korea University Hospital
Seoul 1835848, 02841
South KoreaActive - Recruiting
Samsung Medical Center
Seoul 1835848, 06351
South KoreaActive - Recruiting
Seoul St. Mary's Hospital - The Catholic University of Korea
Seoul 1835848, 065791
South KoreaActive - Recruiting
Severance Hospital
Seoul 1835848, 03722
South KoreaActive - Recruiting
Hospital Universitario Basurto
Bilbao, Bizkaia 48013
SpainSite Not Available
Hospital Universitario Basurto
Bilbao 3128026, Vizcaya 48013
SpainActive - Recruiting
Hospital Clinic de Barcelona
Barcelona, 08036
SpainSite Not Available
Hospital Clinic de Barcelona
Barcelona 3128760, 08036
SpainActive - Recruiting
Hospital General JM Morales Meseguer
Murcia, 30008
SpainSite Not Available
Hospital General JM Morales Meseguer
Murcia 2513416, 30008
SpainActive - Recruiting
Hospital Clinico Universitario de Salamanca
Salamanca 3111108, 37007
SpainActive - Recruiting
China Medical University Hospital
Taichung, Central Taiwan 40447
TaiwanSite Not Available
China Medical University Hospital
Taichung 1668399, Central Taiwan 40447
TaiwanActive - Recruiting
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan, Hunan Province 33305
TaiwanSite Not Available
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan District 1667905, Hunan Province 33305
TaiwanActive - Recruiting
Changhua Christian Hospital
Changhua 1993459, 500-06
TaiwanActive - Recruiting
Changhua Christian Hospital
Changhua City, 500-06
TaiwanSite Not Available
Hualien Tzu Chi Hospital
Hualien City, 97002
TaiwanSite Not Available
Hualien Tzu Chi Hospital
Hualien City 1674504, 97002
TaiwanActive - Recruiting
Kaohsiung Medical University - Chung-Ho Memorial Hospital
Kaohsiung, 807
TaiwanActive - Recruiting
Kaohsiung Medical University Hospital
Kaohsiung, 80756
TaiwanSite Not Available
Kaohsiung Medical University Hospital
Kaohsiung City 1673820, 80756
TaiwanActive - Recruiting
China Medical University Hospital - PPDS
Taichung, 40447
TaiwanActive - Recruiting
Taichung Veterans General Hospital (VGHTC)
Taichung 1668399, 4070
TaiwanActive - Recruiting
Taichung Veterans General Hospital (VGHTC)
Taichung City, 4070
TaiwanSite Not Available
National Cheng Kung University Hospital
Tainan, 704
TaiwanSite Not Available
National Cheng Kung University Hospital
Tainan 1668355, 704
TaiwanActive - Recruiting
National Cheng Kung University Hospital
Tainan City 1668355, 704
TaiwanSite Not Available
National Taiwan University Hospital
Taipei, 100229
TaiwanSite Not Available
Tri-Service General Hospital
Taipei, 11490
TaiwanActive - Recruiting
National Taiwan University Hospital
Taipei 1668341, 100229
TaiwanActive - Recruiting
Tri-Service General Hospital
Taipei 1668341, 114
TaiwanActive - Recruiting
Tri-Service General Hospital
Taipei City, 114
TaiwanSite Not Available
Chang Gung Hospital
Taoyuan City, 33305
TaiwanSite Not Available
National Taiwan University Hospital
Tapei City, 100229
TaiwanSite Not Available
Clinical Research Center, Faculty of Medicine, Prince of Songkla University
Hat Yai 1610780, Changwat Songkhla 1606146 90110
ThailandActive - Recruiting
Rajavithi Hospital
Ratchathewi, Krung Thep Maha Nakhon-Bangkok 10400
ThailandSite Not Available
Clinical Research Center, Faculty of Medicine, Prince of Songkla University
Hat Yai, Songkhla 90110
ThailandSite Not Available
Songklanagarind Hospital Prince of Songkla University
Hat Yai, Songkhla 90110
ThailandActive - Recruiting
King Chulalongkorn Memorial Hospital
Bangkok, 10330
ThailandActive - Recruiting
Rajavithi Hospital
Bangkok, 10400
ThailandSite Not Available
King Chulalongkorn Memorial Hospital
Bangkok 1609350, 10330
ThailandActive - Recruiting
Rajavithi Hospital
Bangkok 1609350, 10400
ThailandSite Not Available
Chaing Mai University
Chiang Mai, 50200
ThailandSite Not Available
Chiang Mai University
Chiang Mai, 50200
ThailandActive - Recruiting
Chaing Mai University
Chiang Mai 1153671, 50200
ThailandActive - Recruiting
Srinagarind Hospital
Khon Kaen, 4002
ThailandSite Not Available
Faculty of Medicine Khon Kaen University
Khon Kaen 1609776, 40002
ThailandActive - Recruiting
Srinagarind Hospital
Khon Kaen 1609776, 40000
ThailandActive - Recruiting
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul, 34418
TurkeyActive - Recruiting
Istanbul University
Istanbul, 34418
TurkeySite Not Available
Ege Universitesi Tip Fakultesi Hastanesi
Izmir, 35100
TurkeyActive - Recruiting
Ege University
Izmir, 35100
TurkeySite Not Available
Istanbul University
Istanbul 745044, 34418
Turkey (Türkiye)Active - Recruiting
Ege University
Izmir 311046, 35100
Turkey (Türkiye)Active - Recruiting
St James Hospital
Leeds, LS9 7TF
United KingdomSite Not Available
St James University Hospital
Leeds, LS9 7TF
United KingdomActive - Recruiting
St James Hospital
Leeds 2644688, LS9 7TF
United KingdomActive - Recruiting
The Oncology Institute of Hope & Innovation
Whittier, California 90603
United StatesSite Not Available
The Oncology Institute of Hope and Innovation
Whittier, California 90603
United StatesActive - Recruiting
The Oncology Institute of Hope & Innovation
Whittier 5409059, California 5332921 90602
United StatesActive - Recruiting

Not the study for you?
Let us help you find the best match. Sign up as a volunteer and receive email notifications when clinical trials are posted in the medical category of interest to you.