Phase
Condition
Hemophilia
Treatment
marstacimab
Clinical Study ID
Ages 1-17 Male
Study Summary
Eligibility Criteria
Inclusion
Inclusion Criteria:
Male participants of appropriate age and required minimum weight
Participants aged 12 to 17 years must be at least 25 kgs at time of consent.
Participants aged 6 to 11 years must be at least 19 kgs at time of consent.
Minimum weight requirement for participants aged 1 to 5 years is to be determined.
Participants with a diagnosis of severe hemophilia A or moderately severe to severehemophilia B
Participants must have at least 1 year of diary and/or medical records available inwhich exogenous FVIII or FIX replacement or bypass agent infusions and hemophilicbleeding episodes were consistently documented over the 12 months prior to the timeof consent.
Participants who are enrolled into the Non-Inhibitor Cohort must also meet the following criteria:
No current detectable inhibitor and no documented history of inhibitors in the 5years prior to consent
Must have at least 50 exposure days to FVIII/FIX replacement products
Must be at least 80% compliant with a stable and effective routine prophylaxisregimen with FVIII/FIX replacement products, for at least 12 months prior to consent
Participants who are enrolled into the Inhibitor Cohort must also meet the following criteria:
Documentation of current high titer inhibitor (≥5 BU/mL); or current low titerinhibitor (<5 BU/mL) refractory to FVIII or FIX replacement and with FVIII or FIXrecovery <60% of expected within previous 12 months prior to the time of consent
Participants who have documented inhibitors while on factor-replacement therapy butwho do not meet the high quantitative inhibitor criteria described in the priorbullet at the time of screening (eg, participant with a previously documentedhigh-titer inhibitor ≥5 BU/mL) and whose condition precludes re-challenge with FVIIIor FIX replacement may be considered for eligibility on a case-by-case basis withdiscussion and agreement from the Pfizer medical monitor.
Hemophilia A participants with on-demand treatment regimen with ≥12 bleedingepisodes or hemophilia B participants with on-demand treatment regimen with ≥8bleeding episodes (spontaneous or traumatic) necessitating treatment with bypassfactor in the 12 months prior to informed consent
Participants must be on an on-demand bypass treatment regimen during the 12 monthsprior to informed consent
Exclusion
Exclusion Criteria:
Known coronary artery, thrombotic, or ischemic disease, or current evidence ofcongenital or acquired thrombophilic disease such as Anti-thrombin III deficiency,Factor V Leiden mutation, prothrombin 20210 mutation, protein C deficiency, proteinS deficiency and antiphospholipid syndrome.
Known planned surgical procedure during the planned study period
Known hemostatic defect other than hemophilia A or B
Abnormal hematology, renal or hepatic function laboratory results at screening
Other acute or chronic medical or psychiatric condition that may increase the riskassociated with study participation or investigational product administration or mayinterfere with the interpretation of study results and, in the judgment of theinvestigator
Individuals with known allergic reaction or hypersensitivity to hamster protein orother components of the study intervention
Current routine prophylaxis with bypassing agent, non-coagulation non-factorreplacement therapy (eg, emicizumab), or any previous treatment with a gene therapyproduct for treatment of hemophilia
Participants with inhibitors who are being treated using a prophylaxis treatmentregimen with a bypass agent, and, participants who have previously receivednon-factor-based hemophilia therapy (eg, fitusiran, concizumab, emicizumab) will beconsidered on a case-by-case basis, only after discussion and agreement between theinvestigator and the Pfizer medical monitor
Regular use of immunomodulatory medications (eg, IVIG, routine systemiccorticosteroids, rituximab)
Use of systemic antifibrinolytics, medications that may increase the risk ofbleeding, and certain non-steroidal anti-inflammatory drugs within 120 hours offirst dose of study intervention and while on study
Ongoing or planned use of ITI, or prophylaxis with FVIII or FIX replacement at anytime after initiation of treatment with study intervention
Participation in other studies involving investigational drug(s) or investigationalvaccine(s) within 30 days (or as determined by local requirements) or 5 half-livesprior to study entry or during study participation
Previous exposure to marstacimab during participation in other marstacimab clinicalstudies
CD4 cell count ≤200/uL if HIV-positive
Abnormal ECG of clinical relevance that may affect participant safety orinterpretation of study results
Investigator site staff members directly involved in the conduct of the study andtheir family members, site staff members otherwise supervised by the investigator,and sponsor and sponsor delegate employees directly involved in the conduct of thestudy and their family members
Study Design
Connect with a study center
Arbesu Hematología
Mendoza, M5501
ArgentinaActive - Recruiting
Sydney Children's Hospital
Randwick, New South Wales 2031
AustraliaSite Not Available
Royal Children's Hospital
Melbourne, Victoria 3052
AustraliaActive - Recruiting
Medizinische Universität Wien
Vienna, Wien 1090
AustriaActive - Recruiting
HEMOES
Vitoria, Espírito Santo 29047-105
BrazilActive - Recruiting
Stollery Children's Hospital
Edmonton, Alberta T6G 2B7
CanadaActive - Recruiting
CancerCare Manitoba
Winnipeg, Manitoba R3E 0V9
CanadaActive - Recruiting
Hamilton Health Sciences - McMaster University Medical Centre
Hamilton, Ontario/canada L8N 3Z5
CanadaActive - Recruiting
Beijing Children's hospital, Capital Medical University
Beijing, Beijing 100045
ChinaActive - Recruiting
Southern Medical University Nanfang Hospital
Guangzhou, Guangdong 510515
ChinaActive - Recruiting
The Affiliated Hospital of Guizhou Medical University
Guiyang, Guizhou 550004
ChinaActive - Recruiting
Tongji Hospital of Tongji Medical College of HUST/Pediatric Hematology Department Pharmacy
Wuhan, Hubei 430030
ChinaActive - Recruiting
Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology
Wuhan, Hubei 430030
ChinaActive - Recruiting
Jiangxi Provincial People's Hospital
Nanchang, Jiangxi 330006
ChinaActive - Recruiting
Institute of hematology&blood disease hospital
Tianjin, Tianjin
ChinaActive - Recruiting
Detska nemocnice FN Brno
Brno, Brno-město 613 00
CzechiaActive - Recruiting
Rigshospitalet
Copenhagen, Hovedstaden 2100
DenmarkActive - Recruiting
Aarhus Universitetshospital, Skejby
Aarhus, Midtjylland 8200
DenmarkSite Not Available
Hôpital Universitaire Necker Enfants Malades
Paris, 75015
FranceActive - Recruiting
Charité Campus Virchow-Klinikum
Berlin, 13353
GermanyActive - Recruiting
Nirmal Hospital Pvt Ltd.
Surat, Gujarat 395002
IndiaActive - Recruiting
K.J. Somaiya Hospital
Mumbai, Maharashtra 400022
IndiaActive - Recruiting
Nil Ratan Sircar Medical College and Hospital
Kolkata, WEST Bengal 700014
IndiaActive - Recruiting
Sheba Medical Center
Ramat Gan, Hamerkaz 5262100
IsraelActive - Recruiting
IRCCS Istituto Giannina Gaslini
Genova, Liguria 16147
ItalyActive - Recruiting
Istituto Clinico Humanitas
Rozzano, Milano 20089
ItalyActive - Recruiting
Ospedale Pediatrico Bambino Gesù IRCCS
Rome, Roma 00165
ItalySite Not Available
Azienda Ospedaliero Universitaria di Parma
Parma, 43126
ItalySite Not Available
Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino
Torino, 10126
ItalySite Not Available
Hyogo prefectural Kobe Children's Hospital
Kobe, Hyogo 650-0047
JapanActive - Recruiting
Nagano Children's Hospital
Azumino, Nagano 399-8288
JapanActive - Recruiting
Nara Medical University Hospital
Kashihara, Nara 634-8522
JapanActive - Recruiting
Saitama Prefectural Children's Medical Center
Saitama-shi, Saitama 330-8777
JapanActive - Recruiting
Saga University Hospital
Saga, 849-8501
JapanActive - Recruiting
Kyung Hee University Hospital at Gangdong
Seoul, Seoul-teukbyeolsi [seoul] 05278
Korea, Republic ofActive - Recruiting
Severance Hospital, Yonsei University Health System
Seoul, Seoul-teukbyeolsi [seoul] 03722
Korea, Republic ofActive - Recruiting
Kyungpook National University Hospital
Daegu, Taegu-kwangyǒkshi 41944
Korea, Republic ofActive - Recruiting
Arké SMO S.A de C.V
Veracruz, 91910
MexicoSite Not Available
King Fahad Specialist Hospital - Dammam
Dammam, ASH Sharqīyah 31444
Saudi ArabiaSite Not Available
King Faisal Specialist Hospital & Research Center
An Narjis,
Saudi ArabiaSite Not Available
King Faisal Specialist Hospital & Research Center
An Narjis, Riyadh,
Saudi ArabiaSite Not Available
King Faisal Specialist Hospital & Research Center
Riyadh,
Saudi ArabiaSite Not Available
Univerzitna nemocnica Bratislava, Nemocnica sv. Cyrila a Metoda
Bratislava, 851 07
SlovakiaActive - Recruiting
Detska fakultna nemocnica Kosice
Kosice, 040 11
SlovakiaActive - Recruiting
Univerzitna nemocnica Martin
Martin, 036 59
SlovakiaActive - Recruiting
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Benoni, Gauteng 1500
South AfricaSite Not Available
Charlotte Maxeke Johannesburg Academic Hospital
Johannesburg, Gauteng 2193
South AfricaSite Not Available
Haemophilia Comprehensive Care Centre
Johannesburg, Gauteng 2193
South AfricaSite Not Available
Hospital Universitario La Paz
Madrid, 28046
SpainActive - Recruiting
Hospital Universitario Miguel Servet
Zaragoza, 50009
SpainActive - Recruiting
Changhua Christian Hospital
Changhua County, Changhua 50006
TaiwanActive - Recruiting
Taichung Veterans General Hospital
Taichung, 407
TaiwanActive - Recruiting
National Taiwan University Hospital
Taipei, 10002
TaiwanActive - Recruiting
Erciyes University
Talas, Kayseri 38039
TurkeyActive - Recruiting
Istanbul Universitesi Istanbul Tıp Fakultesi Hastanesi
İstanbul, İ̇stanbul 34093
TurkeyActive - Recruiting
Ege Universitesi Hastanesi
Izmir, İ̇zmir 35100
TurkeyActive - Recruiting
Acibadem Adana Hospital
Adana, 01130
TurkeyActive - Recruiting
Gazi University Health Research and Application Center Gazi Hospital
Ankara, 06560
TurkeyActive - Recruiting
Erciyes Universitesi Tıp Fakultesi Hastaneleri
Kayseri, 38039
TurkeyActive - Recruiting
Ondokuz Mayıs Universitesi
Samsun, 55200
TurkeyActive - Recruiting
Royal Victoria Infirmary
Newcastle upon Tyne, England NE1 4LP
United KingdomActive - Recruiting
Birmingham Children's Hospital
Birmingham, B4 6NH
United KingdomSite Not Available
Leeds General Infirmary
Leeds, LS1 3EX
United KingdomSite Not Available
Evelina London Children's Hospital
London, SE1 7EH
United KingdomActive - Recruiting
Royal Manchester Children's Hospital
Manchester, M13 9WL
United KingdomActive - Recruiting
Intermountain - Primary Children's Hospital
Salt Lake City, Utah 84113
United StatesActive - Recruiting
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