Actively Recruiting
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
Eligibility Criteria
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
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
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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
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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