Phase
Condition
Hemoglobinuria, Paroxysmal
Aplastic Anemia
Red Blood Cell Disorders
Treatment
Eculizumab
Ravulizumab
Cemdisiran
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 vaccination is less than 2 weeks prior tostudy treatment initiation)
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
Toronto General Hospital
Toronto, Ontario M5G2C4
CanadaActive - Recruiting
Peking Union Medical College Hospital
Beijing, Beijing 100032
ChinaActive - Recruiting
Hospital Pablo Tobon Uribe
Medellin, Antioquia 050034
ColombiaActive - Recruiting
George Papanikolaou Hospital
Thessaloniki, 57010
GreeceActive - Recruiting
Semmelweis Egyetem
Budapest, 1089
HungaryActive - Recruiting
Semmelweis University
Budapest, 1083
HungaryActive - Recruiting
Rajiv Gandhi Cancer Institute & Research Center (RGCIRC) - Rohini Campus
New Delhi, Delhi 110085
IndiaActive - Recruiting
Rajiv Gandhi Cancer Institute and Research Centre
New Delhi, Delhi 110085
IndiaActive - Recruiting
Malabar Cancer Center, Kerala
Kannur, Kerala 670103
IndiaActive - Recruiting
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 855972
IndiaActive - Recruiting
K J Somaiya Super Specialty Hospital & Research Centre
Mumbai, Maharashtra 400022
IndiaActive - Recruiting
Postgraduate Institute of Medical Education & Research (PGIMER)
Chandigarh, Punjab 708248
IndiaActive - Recruiting
Bhagwan Mahaveer Cancer Hospital & Research Centre
Jaipur, Rajasthan 302017
IndiaActive - Recruiting
Kings George's Medical University
Lucknow, Uttar Pradesh 226014
IndiaSite Not Available
Postgraduate Institute of Medical Education and Research
Chandigarh, 160012
IndiaActive - Recruiting
Bhagwan Mahaveer Cancer Hospital and Research Centre (BMCHRC)
Jaipur, 302017
IndiaActive - Recruiting
Malabar Cancer Center, Kerala
Kerala, 670103
IndiaSite Not Available
King George Hospital
Uttar Pradesh, 226003
IndiaActive - Recruiting
Aou Careggi
Firenze, Forence 50139
ItalyActive - Recruiting
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
Azienda Ospedaliera Universitaria Careggi
Firenze, 50134
ItalyActive - Recruiting
Fondazione Policlinico Universitrio a. Gemelli - IRCCS
Rome, 000168
ItalyActive - Recruiting
Hematology Citta della Salute e della Scienza di Torino
Turin, 10126
ItalyActive - Recruiting
Japanese Red Cross Aichi Medical Center Nagoya Daini Hospital
Nagoya, Aiti 466-8650
JapanActive - Recruiting
Ogaki Municipal Hospital
Ogaki, Gifu 503-8502
JapanActive - Recruiting
Ogaki Municipal Hospital
Ogaki city, Gifu 503-8502
JapanSite Not Available
University of Tsukuba Hospital
Tsukuba, Ibaraki 305-8576
JapanActive - Recruiting
University of Tsukuba Hospital
Tsukuba-shi, Ibaraki 305-8576
JapanSite Not Available
Matsushita Memorial Hospital
Moriguchi, Osaka 570-8540
JapanActive - Recruiting
Panasonic Health Insurance Organization Matsushita Memorial Hospital
Moriguchi-city, Osaka 570-8540
JapanSite Not Available
NTT Medical Center Tokyo
Shinagawa-ku, Tokyo 141-8625
JapanActive - Recruiting
Jordan University Hospital (JUH)
Amman, 11942
JordanActive - Recruiting
University of Jordan
Amman, 11196
JordanActive - Recruiting
St. Vincent Hospital
Suwon-si, Gyeonggi-do 16247
Korea, Republic ofActive - Recruiting
Gachon University Gil Medical Center
Incheon, Namdong-Gu 21565
Korea, Republic ofActive - Recruiting
Pusan National University Hospital
Busan, 49241
Korea, Republic ofActive - Recruiting
Ajou University Medical Center
Gyeonggi-do, 16499
Korea, Republic ofSite Not Available
Ajou University Medical Center
Gyeonggido, 16499
Korea, Republic ofActive - Recruiting
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 ofActive - Recruiting
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 Ampang
Ampang, Pahang 68000
MalaysiaActive - Recruiting
Hospital Tengku Ampuan Afzan
Kuantan, Pahang 25200
MalaysiaActive - Recruiting
Hospital Tg Ampuan Afzan
Kuantan, Pahang 25100
MalaysiaActive - Recruiting
Hospital Queen Elizabeth
Kota Kinabalu, Sabah 88586
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
MexicoActive - Recruiting
Hospital Universitario Dr. Jose Eleuterio González
Monterrey, Nuevo León 64460
MexicoActive - Recruiting
Clinica San Felipe
Lima, 15072
PeruActive - Recruiting
St Lukes Medical Center
Quezon City, Central Luzon 1011
PhilippinesActive - Recruiting
University Clinical Center Medical University of Gdansk
Gdansk, Pomeranian Voivodeship 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
PolandActive - Recruiting
Uniwersyteckie Centrum Kliniczne
Gdansk, 80-214
PolandActive - Recruiting
Institute of Hematology and Transfusion Medicine
Warsaw, 02-776
PolandActive - Recruiting
Instytut Hematologii i Transfuzjologii
Warszawa, 02-776
PolandSite Not Available
Ion Chiricuta Oncology Institute
Cluj-Napoca, Cluj 400124
RomaniaActive - Recruiting
Prof Dr I Chiricuta Institute of Oncology
Cluj-Napoca, Cluj 400015
RomaniaActive - Recruiting
Filantropia Municipal Clinical Hospital
Craiova, Dolj 200143
RomaniaActive - Recruiting
Municipal Hospital Filantropia
Craiova, Dolj 200143
RomaniaActive - Recruiting
Targu Mures Clinical County Emergency Hospital
Targu Mures, Mures 540136
RomaniaActive - Recruiting
Targu-Mures Emergency Clinical County Hospital
Targu Mures, Mures 540136
RomaniaActive - Recruiting
National University Hospital
Singapore, 119074
SingaporeActive - Recruiting
Hospital Universitario Basurto
Bilbao, Bizkaia 48013
SpainActive - Recruiting
Hospital Clinic de Barcelona
Barcelona, 08036
SpainActive - Recruiting
China Medical University Hospital
Taichung, Central Taiwan 40447
TaiwanActive - Recruiting
Chang Gung Memorial Hospital - Linkou Branch
Taoyuan, Hunan Province 333
TaiwanActive - Recruiting
Changhua Christian Hospital
Changhua City, 500-06
TaiwanActive - Recruiting
Hualien Tzu Chi Hospital
Hualien City, 97002
TaiwanActive - Recruiting
Kaohsiung Medical University - Chung-Ho Memorial Hospital
Kaohsiung, 807
TaiwanActive - Recruiting
Kaohsiung Medical University Hospital
Kaohsiung, 80756
TaiwanActive - Recruiting
China Medical University Hospital - PPDS
Taichung, 40447
TaiwanActive - Recruiting
Taichung Veterans General Hospital (VGHTC)
Taichung City, 4070
TaiwanActive - Recruiting
National Cheng Kung University Hospital
Tainan, 704
TaiwanActive - Recruiting
National Taiwan University Hospital
Taipei, 100229
TaiwanActive - Recruiting
Tri-Service General Hospital
Taipei, 11490
TaiwanActive - Recruiting
Tri-Service General Hospital
Taipei City, 114
TaiwanActive - Recruiting
Chang Gung Hospital
Taoyuan City, 33305
TaiwanSite Not Available
National Taiwan University Hospital
Tapei City, 100229
TaiwanSite Not Available
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
ThailandActive - Recruiting
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
Chaing Mai University
Chiang Mai, 50200
ThailandActive - Recruiting
Chiang Mai University
Chiang Mai, 50200
ThailandActive - Recruiting
Srinagarind Hospital
Khon Kaen, 4002
ThailandActive - Recruiting
Istanbul Universitesi Istanbul Tip Fakultesi Hastanesi
Istanbul, 34418
TurkeyActive - Recruiting
Istanbul University
Istanbul, 34418
TurkeyActive - Recruiting
Ege Universitesi Tip Fakultesi Hastanesi
Izmir, 35100
TurkeyActive - Recruiting
Ege University
Izmir, 35100
TurkeyActive - Recruiting
St James Hospital
Leeds, LS9 7TF
United KingdomActive - Recruiting
St James University Hospital
Leeds, LS9 7TF
United KingdomActive - Recruiting
The Oncology Institute of Hope & Innovation
Whittier, California 90603
United StatesActive - Recruiting
The Oncology Institute of Hope and Innovation
Whittier, California 90603
United StatesActive - Recruiting
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