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New treatment option for Hemophilia A
Official title A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
ClinicalTrials.gov ID: NCT05611801
What this study is testing
What is marstacimab?
marstacimab is an investigational medicine, given as an once-weekly injection under the skin, being studied as a potential treatment for hemophilia a.
Also referred to as PF-06741086.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 3: a large, late-stage study
- Time commitment: about 14 months
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 17, men only
You may be able to join if
- 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
You likely can't join if
- Known coronary artery, thrombotic, or ischemic disease, or current evidence of congenital or acquired thrombophilic disease such as Anti-thrombin III...
- 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 risk associated with study participation or investigational product...
- Individuals with known allergic reaction or hypersensitivity to hamster protein or other components of the study intervention
See the full eligibility 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 severe hemophilia B
- Participants must have at least 1 year of diary and/or medical records available in which exogenous FVIII or FIX replacement or bypass agent infusions and hemophilic bleeding episodes were consistently documented over...
- No current detectable inhibitor and no documented history of inhibitors in the 5 years 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 prophylaxis regimen with FVIII/FIX replacement products, for at least 12 months prior to consent Participants who are enrolled into the Inhibitor Cohort...
- Documentation of current high titer inhibitor (≥5 BU/mL); or current low titer inhibitor (\<5 BU/mL) refractory to FVIII or FIX replacement and with FVIII or FIX recovery \<60% of expected within previous 12 months...
- Participants who have documented inhibitors while on factor-replacement therapy but who do not meet the high quantitative inhibitor criteria described in the prior bullet at the time of screening (eg, participant with a...
- Hemophilia A participants with on-demand treatment regimen with ≥12 bleeding episodes or hemophilia B participants with on-demand treatment regimen with ≥8 bleeding episodes (spontaneous or traumatic) necessitating...
- Participants must be on an on-demand bypass treatment regimen during the 12 months prior to informed consent
- Known coronary artery, thrombotic, or ischemic disease, or current evidence of congenital or acquired thrombophilic disease such as Anti-thrombin III deficiency, Factor V Leiden mutation, prothrombin 20210 mutation...
- 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 risk associated with study participation or investigational product administration or may interfere with the interpretation of study results...
- Individuals with known allergic reaction or hypersensitivity to hamster protein or other components of the study intervention
- Current routine prophylaxis with bypassing agent, non-coagulation non-factor replacement therapy (eg, emicizumab), or any previous treatment with a gene therapy product for treatment of hemophilia
- Participants with inhibitors who are being treated using a prophylaxis treatment regimen with a bypass agent, and, participants who have previously received non-factor-based hemophilia therapy (eg, fitusiran...
- Regular use of immunomodulatory medications (eg, IVIG, routine systemic corticosteroids, rituximab)
- Use of systemic antifibrinolytics, medications that may increase the risk of bleeding, and certain non-steroidal anti-inflammatory drugs within 120 hours of first dose of study intervention and while on study
- Ongoing or planned use of ITI, or prophylaxis with FVIII or FIX replacement at any time after initiation of treatment with study intervention
- Participation in other studies involving investigational drug(s) or investigational vaccine(s) within 30 days (or as determined by local requirements) or 5 half-lives prior to study entry or during study participation
- Previous exposure to marstacimab during participation in other marstacimab clinical studies
- CD4 cell count ≤200/uL if HIV-positive
- Abnormal ECG of clinical relevance that may affect participant safety or interpretation of study results
- Investigator site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the investigator, and sponsor and sponsor delegate employees directly...
The study team makes the final eligibility decision.
Where it's taking place
- Mendoza, Argentina
- Parkville, Australia
- Vienna, State of Vienna, Austria
- Hamilton, Ontario/canada, Canada
- Wuhan, Hubei, China
- Tianjin, Tianjin Municipality, China
- Brno, Brno-město, Czechia
- Prague, Czechia
- Copenhagen, Capital Region, Denmark
- Aarhus N, Denmark
- Berlin, Germany
- Rome, ROMA, Italy
- Parma, Italy
- Torino, Italy
- Kobe, Hyōgo, Japan
- Saitama-shi, Saitama, Japan
- Saga, Japan
- Veracruz, Mexico
- Dammam, Saudi Arabia
- Košice, Slovakia
+ 12 more site(s).
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The study runs about 14 months per participant, based on its public description. The team confirms the exact schedule and number of visits before you enroll.
Who can join this trial?
This study is enrolling male, 1 year to 17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Mendoza, Argentina; Parkville, Australia; Vienna, State of Vienna, Austria; Hamilton, Ontario/canada, Canada; Wuhan, Hubei, China; Tianjin, Tianjin Municipality, China and 26 more location(s). Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.