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.
Study Type
INTERVENTIONAL
Allocation
NA
Purpose
TREATMENT
Masking
NONE
Enrollment
100
marstacimab
Intermountain - Primary Children's Hospital
Salt Lake City, Utah, United States
RECRUITINGArbesu Hematología
Mendoza, Argentina
RECRUITINGSydney Children's Hospital
Randwick, New South Wales, Australia
NOT_YET_RECRUITINGRoyal Children's Hospital
Melbourne, Victoria, Australia
Annualized bleeding rate (ABR) of treated bleeding events
Derived for each subject for each period (historical and study treatment) by using the following formula: ABR = number of bleeds requiring treatments/ (days on treatment period / 365.25)
Time frame: Baseline to end of 12-month treatment period
Incidence of adverse events and serious adverse events
Time frame: Screening through end of follow-up period (approximately 14 months)
Incidence and severity of thrombotic events
Time frame: Baseline to end of 12-month treatment period
Incidence and severity of thrombotic microangiopathy
Time frame: Baseline to end of 12-month treatment period
Incidence and severity of disseminated intravascular coagulation/consumption coagulopathy events
Time frame: Baseline to end of 12-month treatment period
Immunogenicity (incidence of ADA and clinically significant persistent NAb against marstacimab)
Time frame: Baseline to end of 12-month treatment period
Incidence and severity of injection site reaction
Time frame: Baseline to end of 12-month treatment period
Incidence of severe hypersensitivity and anaphylactic reactions
Time frame: Baseline to end of 12-month treatment period
Incidence of joint bleeds (treated)
Time frame: Baseline to end of 12-month treatment period
Incidence of spontaneous bleeds (treated)
Time frame: Baseline to end of 12-month treatment period
Incidence of target joint bleeds (treated)
Time frame: Baseline to end of 12-month treatment period
Incidence of total bleeds (treated and untreated)
Time frame: Baseline to end of 12-month treatment period
Number of target joints
Time frame: Baseline to end of 12-month treatment period
Change from baseline in joint health as measured by the HJHS for participants ≥4 years of age
Time frame: Baseline to end of 12-month treatment period
Changes in quality of life measured by Haem-A-QoL/Haemo-QoL (using age-dependent versions for participants ≥8 years of age)
Time frame: Baseline to end of 12-month treatment period
Changes in quality of life measured by pedHAL (using age-dependent versions for participants ≥4 years of age)
Time frame: Baseline to end of 12-month treatment period
Changes in quality of life measured by Patient Global Impression of Change - Hemophilia for participants ≥4 years of age
Time frame: Baseline to end of 12-month treatment period
Changes in quality of life measured by Health Utilities Measure (EQ-5D-Y) for participants ≥4 years of age
Time frame: Baseline to end of 12-month treatment period
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Medizinische Universität Wien
Vienna, Vienna, Austria
RECRUITINGHEMOES
Vitória, Espírito Santo, Brazil
RECRUITINGStollery Children's Hospital
Edmonton, Alberta, Canada
RECRUITINGHamilton Health Sciences - McMaster University Medical Centre
Hamilton, Ontario/canada, Canada
RECRUITINGBeijing Children's hospital, Capital Medical University
Beijing, Beijing Municipality, China
RECRUITINGSouthern Medical University Nanfang Hospital
Guangzhou, Guangdong, China
RECRUITING...and 50 more locations