Actively Recruiting

Phase 3
Age: 1Year - 17Years
MALE
NCT05611801

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

Led by Pfizer · Updated on 2026-05-07

100

Participants Needed

34

Research Sites

300 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

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.

CONDITIONS

Official Title

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

Who Can Participate

Age: 1Year - 17Years
MALE

Eligibility Criteria

Eligible

You may qualify if you...

  • Male participants aged 1 to 17 years
  • Participants aged 12 to 17 years must weigh at least 25 kg
  • Participants aged 6 to 11 years must weigh at least 19 kg
  • Weight requirement for participants aged 1 to 5 years to be determined
  • Diagnosis of severe hemophilia A or moderately severe to severe hemophilia B
  • At least 1 year of records documenting factor infusions and bleeding episodes
  • Non-inhibitor patients must have no inhibitors in past 5 years
  • Non-inhibitor patients must have at least 50 exposure days to factor replacement
  • Non-inhibitor patients must be at least 80% compliant with routine prophylaxis for 12 months
  • Inhibitor patients must have current high or refractory low titer inhibitor
  • Inhibitor patients must have had specified bleeding episodes requiring bypass treatment in past 12 months
  • Inhibitor patients must have been on on-demand bypass treatment regimen during past 12 months
Not Eligible

You will not qualify if you...

  • Known coronary artery, thrombotic, or ischemic disease or thrombophilic conditions
  • Planned surgery during the study period
  • Known bleeding disorder other than hemophilia A or B
  • Abnormal blood, kidney, or liver lab results at screening
  • Acute or chronic medical or psychiatric conditions increasing risk or interfering with results
  • Known allergy or hypersensitivity to hamster protein or study components
  • Current routine prophylaxis with bypassing agents or non-factor therapies
  • Previous gene therapy for hemophilia
  • Inhibitor patients on prophylaxis with bypass agents or prior non-factor therapies without agreement
  • Regular use of immunomodulatory medications
  • Use of antifibrinolytics, medications increasing bleeding risk, or certain NSAIDs within 120 hours before first dose and during study
  • Planned or ongoing immune tolerance induction or prophylaxis with factor replacement after starting study drug
  • Participation in other investigational drug or vaccine studies within 30 days or 5 half-lives before or during study
  • Previous exposure to marstacimab in other studies
  • CD4 count ≤200/uL if HIV-positive
  • Clinically relevant abnormal ECG
  • Investigator site staff involved in study and their family members, or sponsor employees and their family members involved in study conduct

AI-Screening

AI-Powered Screening

Complete this quick 3-step screening to check your eligibility

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Trial Site Locations

Total: 34 locations

1

Arbesu Hematología

Mendoza, Argentina, M5501

Actively Recruiting

2

Murdoch Children's Research Institute

Parkville, Australia, 3052

Actively Recruiting

3

Medizinische Universität Wien

Vienna, State of Vienna, Austria, 1090

Actively Recruiting

4

Hamilton Health Sciences - McMaster University Medical Centre

Hamilton, Ontario/canada, Canada, L8N 3Z5

Actively Recruiting

5

Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology

Wuhan, Hubei, China, 430030

Actively Recruiting

6

Tongji Hospital of Tongji Medical College of HUST/Pediatric Hematology Department Pharmacy

Wuhan, Hubei, China, 430030

Actively Recruiting

7

Institute of hematology&blood disease hospital

Tianjin, Tianjin Municipality, China

Actively Recruiting

8

Detska nemocnice FN Brno

Brno, Brno-město, Czechia, 613 00

Actively Recruiting

9

Motol University Hospital

Prague, Czechia, 150 06

Actively Recruiting

10

Rigshospitalet

Copenhagen, Capital Region, Denmark, 2100

Actively Recruiting

11

Aarhus Universitetshospital, Skejby

Aarhus N, Denmark, 8200

Actively Recruiting

12

Charité Campus Virchow-Klinikum

Berlin, Germany, 13353

Actively Recruiting

13

Ospedale Pediatrico Bambino Gesù IRCCS

Rome, ROMA, Italy, 00165

Actively Recruiting

14

Azienda Ospedaliero Universitaria di Parma

Parma, Italy, 43126

Actively Recruiting

15

Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino

Torino, Italy, 10126

Not Yet Recruiting

16

Hyogo prefectural Kobe Children's Hospital

Kobe, Hyōgo, Japan, 650-0047

Actively Recruiting

17

Saitama Prefectural Children's Medical Center

Saitama-shi, Saitama, Japan, 330-8777

Actively Recruiting

18

Saga University Hospital

Saga, Japan, 849-8501

Actively Recruiting

19

Arké SMO S.A de C.V

Veracruz, Mexico, 91900

Not Yet Recruiting

20

King Fahad Specialist Hospital

Dammam, Saudi Arabia, 32253

Actively Recruiting

21

Detska fakultna nemocnica Kosice

Košice, Slovakia, 040 11

Actively Recruiting

22

Severance Hospital, Yonsei University Health System

Seoul, Seoul-teukbyeolsi [seoul], South Korea, 03722

Actively Recruiting

23

Kyung Hee University Hospital at Gangdong

Seoul, Seoul-teukbyeolsi [seoul], South Korea, 05278

Actively Recruiting

24

Hospital Universitario La Paz

Madrid, Madrid, Comunidad de, Spain, 28046

Actively Recruiting

25

Changhua Christian Hospital

Changhua County, Changhua, Taiwan, 50006

Actively Recruiting

26

Taichung Veterans General Hospital

Taichung, Taiwan, 407219

Actively Recruiting

27

National Taiwan University Hospital

Taipei, Taiwan, 100

Actively Recruiting

28

Gazi Universitesi Tip Fakultesi Hastanesi Cocuk Hematologi Bolumu

Ankara, Turkey (Türkiye), 06560

Actively Recruiting

29

Ege University

Izmir, Turkey (Türkiye), 35010

Actively Recruiting

30

Erciyes University Faculty of Medicine Pediatric

Kayseri, Turkey (Türkiye), 38039

Actively Recruiting

31

Ondokuz Mayıs Universitesi

Samsun, Turkey (Türkiye), 55200

Actively Recruiting

32

Royal Victoria Infirmary

Newcastle upon Tyne, England, United Kingdom, NE1 4LP

Actively Recruiting

33

Royal Manchester Children's Hospital

Manchester, United Kingdom, M13 9WL

Actively Recruiting

34

Freeman Hospital

Newcastle upon Tyne, United Kingdom, NE7 7DN

Actively Recruiting

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Research Team

P

Pfizer CT.gov Call Center

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NA

Model

SINGLE_GROUP

Primary Purpose

TREATMENT

Number of Arms

1

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