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Looking for participantsPhase3

A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B

Sponsor: Pfizer

NCT ID: NCT05611801

View on ClinicalTrials.gov ↗

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.

Hemophilia AHemophilia B

How this trial compares with your answers

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What we know so far

Condition· Matched your search
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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

CD4

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
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 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 the 12 months prior to the time of 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 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 must also meet the following criteria: * 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 prior to the time of consent * 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 previously documented high-titer inhibitor ≥5 BU/mL) and whose condition precludes re-challenge with FVIII or FIX replacement may be considered for eligibility on a case-by-case basis with discussion and agreement from the Pfizer medical monitor. * 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 treatment with bypass factor in the 12 months prior to informed consent * Participants must be on an on-demand bypass treatment regimen during the 12 months prior to informed consent Exclusion Criteria: * 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, protein C deficiency, protein S 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 risk associated with study participation or investigational product administration or may interfere with the interpretation of study results and, in the judgment of the investigator * 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, concizumab, emicizumab) will be considered on a case-by-case basis, only after discussion and agreement between the investigator and the Pfizer medical monitor * 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 involved in the conduct of the study and their family members

Where Is This Study? (5 UK sites)

Royal Victoria Infirmary

Newcastle upon Tyne NE1 4LP, United Kingdom

Recruiting
Hospital R&D contact (matched)

Colleen Bowthorpe

colleen.bowthorpe@nhs.scot01387 241815

Birmingham Children's Hospital

Birmingham B4 6NH, United Kingdom

Recruiting
Hospital R&D contact (matched)

Sarah Pountain Head of Research Governance

R&D@uhb.nhs.uk0121 371 4185

Evelina London Children's Hospital

London SE1 7EH, United Kingdom

Recruiting

Royal Manchester Children's Hospital

Manchester M13 9WL, United Kingdom

Recruiting
Hospital R&D contact (matched)

Elizabeth Mainwaring

R&D.applications@mft.nhs.uk0161 276 3340

Freeman Hospital

Newcastle upon Tyne NE7 7DN, United Kingdom

Recruiting

How to Get in Touch

Pfizer CT.gov Call Center

Sponsor contact

CONTACT

1-800-718-1021 ClinicalTrials.gov_Inquiries@pfizer.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-09