At a glance
- What the study gets you
- Access to the study treatment being tested
- Type of study
- Interventional (receives a drug or procedure)
- Time in hospital
- In-person visits at study sites — visit count not specified by the sponsor
- Drug or intervention
- marstacimab (drug)
- How long the study runs
- Study runs about 69 months (dates as stated)
- About the drug or intervention
- marstacimab — drug: marstacimab
- Patient visit burden
- Not specified by the sponsor
- Type of study
- Testing a treatment
- Ages
- 1 Year to 17 Years
- Who
- Male
- Number of participants
- 100
- Started
- 2022-12-09
- Last checked
- 2026-09
Plain English Summary
What is this study?
- • Testing a new treatment for hemophilia a
- • Phase3 - 100 participants
- • 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
Who can take part?
- • Ages 1 Year to 17 Years
- • Diagnosed with hemophilia a
- • Male only
Where?
- • Newcastle upon Tyne - Royal Victoria Infirmary
- • Birmingham - Birmingham Children's Hospital
- • London - Evelina London Children's Hospital
- • Manchester - Royal Manchester Children's Hospital
- • +1 more UK sites
This is a simplified summary. Always discuss with your doctor before making any decisions.
About This Trial
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.
How this trial compares with your answers
Answer 2 more questions to improve match
What we know so far
Still need:
- • Tell us your age for better matching
- • Tell us your sex for better matching
Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.
Eligibility at a Glance
Key info
- Age: 1 Year - 17 Years
- Who can join: Male only
Biomarkers mentioned
Treatment history
Treatments you must have had:
- ✓ minimum weight
- ✓ at least 50 exposure days to FVIII/FIX replacement products
What the study is looking for
- ✓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 severe hemophilia B
Who cannot take part
- ✗Known planned surgical procedure during the planned study period
- ✗Known hemostatic defect other than hemophilia A or B
- ✗Abnormal hematology, kidney or liver function laboratory results at screening
- ✗Individuals with known allergic reaction or hypersensitivity to hamster protein or other components of the study...
- ✗Current routine prophylaxis with bypassing agent, non-coagulation non-factor replacement therapy (eg, emicizumab),...
See the full criteria
Where Is This Study? (5 UK sites)
Royal Victoria Infirmary
Newcastle upon Tyne NE1 4LP, United Kingdom
Birmingham Children's Hospital
Birmingham B4 6NH, United Kingdom
Evelina London Children's Hospital
London SE1 7EH, United Kingdom
Royal Manchester Children's Hospital
Manchester M13 9WL, United Kingdom
Freeman Hospital
Newcastle upon Tyne NE7 7DN, United Kingdom
How to Get in Touch
Pfizer CT.gov Call Center
Sponsor contactCONTACT
