研究单位：[1]Pfizer[2],Korea, Republic of,03722[3],Korea, Republic of,05278[4]Sydney Children's Hospital,Randwick,New South Wales,Australia,2031[5]Royal Children's Hospital,Melbourne,Victoria,Australia,3052[6]HEMOES,Vitoria,Espírito Santo,Brazil,29047-105[7]Stollery Children's Hospital,Edmonton,Alberta,Canada,T6G 2B7[8]CancerCare Manitoba,Winnipeg,Manitoba,Canada,R3E 0V9[9]Hamilton Health Sciences - McMaster University Medical Centre,Hamilton,Ontario/canada,Canada,L8N 3Z5[10]Beijing Children's hospital, Capital Medical University,Beijing,Beijing,China,100045[11]Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology,Wuhan,Hubei,China,430030[12]Tongji Hospital of Tongji Medical College of HUST/Pediatric Hematology Department Pharmacy,Wuhan,Hubei,China,430030[13]Jiangxi Provincial People's Hospital,Nanchang,Jiangxi,China,330006[14]Institute of hematology&blood disease hospital,Tianjin,Tianjin,China,300020[15]Nirmal Hospital Pvt Ltd.,Surat,Gujarat,India,395002[16]K.J. Somaiya Hospital,Mumbai,Maharashtra,India,400022[17]Nil Ratan Sircar Medical College and Hospital,Kolkata,WEST Bengal,India,700014[18]Sheba Medical Center,Ramat Gan,Hamerkaz,Israel,5262100[19]Hyogo prefectural Kobe Children's Hospital,Kobe,Hyogo,Japan,650-0047[20]Hyogo prefectural Kobe Children's Hospital,Kobe,Hyogo,Japan,650-0047[21]Nagano Children's Hospital,Azumino,Nagano,Japan,399-8288[22]Saitama Prefectural Children's Medical Center,Saitama-shi,Saitama,Japan,330-8777[23]Saga University Hospital,Saga,Japan,849-8501[24]Kyungpook National University Hospital,Daegu,Taegu-kwangyǒkshi,Korea, Republic of,41944[25]King Faisal Specialist Hospital & Research Center,An Narjis, Riyadh,Saudi Arabia[26]King Faisal Specialist Hospital & Research Center,Riyadh,Saudi Arabia[27]Worthwhile Clinical Trials,Benoni,Gauteng,South Africa,1500[28]Charlotte Maxeke Johannesburg Academic Hospital,Johannesburg,Gauteng,South Africa,2193[29]Changhua Christian Hospital,Changhua County,Changhua,Taiwan,50006[30]Taichung Veterans General Hospital,Taichung,Taiwan,40705[31]Taichung Veterans General Hospital,Taichung,Taiwan,407[32]National Taiwan University Hospital,Taipei,Taiwan,10002[33]Erciyes University,Talas,Kayseri,Turkey,38039[34]Acibadem Adana Hospital,Adana,Turkey,01130[35]Gazi University Health Research and Application Center Gazi Hospital,Ankara,Turkey,06560[36]Erciyes Universitesi Tıp Fakultesi Hastaneleri,Kayseri,Turkey,38039[37]Ondokuz Mayıs Universitesi,Samsun,Turkey,55200
The purpose of this clinical trial is to learn about the safety and effects of the study medicine (called marstacimab) for the potential treatment of hemophilia in pediatric patients. This study will enroll pediatric participants from ages 1 to 17 years in a sequential manner. The study will open enrollment to adolescent participants aged 12 to 17 years first. Then children aged 6 to 11 years will be permitted to enroll. Lastly, children aged 1 to 5 years will be permitted to enroll. This study will enroll participants who: - have severe Hemophilia A or moderately severe to severe Hemophilia B (with or without inhibitors) - have accurate historical records documenting all factor VIII, factor IX, or bypass agent infusions and hemophilia bleed events for at least 1 year prior to entering the study - if a non-inhibitor patient, must be on a stable routine prophylaxis regimen with factor VIII or factor IX replacement products for at least 12 months prior to study entry - if an inhibitor patient, must be on an on-demand bypass treatment regimen during the 12 months prior to study entry All participants in this study will receive marstacimab to use prophylactically. Marstacimab will be given once a week as a subcutaneous (under the skin) shot. The first dose of marstacimab will be given at the study site by the study site staff. During the 12-month treatment period, weekly doses of marstacimab can be given at home, or if preferred, the doses may be given by the study site staff. To help us determine if the study medicine is safe and effective, we will compare participant experiences when they are taking the study medicine to a historical period when they were not. Researchers want to see if the study medicine works to prevent the bleeding episodes commonly experienced by patients with Hemophilia. Participants will be in this study for about 14 months (approximately 1 month in a Screening period, 12 months receiving treatment, and 1 month in a follow-up period) during which they will visit the study site at least 10 times. If preferred, and if local regulations allow it, 2 of the study visits can be completed at the participant's home instead of at the study site. There will also be 6 scheduled telephone calls approximately every 2 months.
