A Clinical Trial of Study Medicine (Marstacimab) in Pediatric Patients With Hemophilia A or Hemophilia B
Condition(s) studied
Study summary
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.
Eligibility
Primary outcome measure(s)
- Annualized bleeding rate (ABR) of treated bleeding events — Baseline to end of 12-month treatment period
Derived for each subject for each period (historical and study treatment) by using the following formula: ABR = number of bleeds requiring treatments/ (days on treatment period / 365.25) - Incidence of adverse events and serious adverse events — Screening through end of follow-up period (approximately 14 months)
- Incidence and severity of thrombotic events — Baseline to end of 12-month treatment period
- Incidence and severity of thrombotic microangiopathy — Baseline to end of 12-month treatment period
- Incidence and severity of disseminated intravascular coagulation/consumption coagulopathy events — Baseline to end of 12-month treatment period
- Immunogenicity (incidence of ADA and clinically significant persistent NAb against marstacimab) — Baseline to end of 12-month treatment period
- Incidence and severity of injection site reaction — Baseline to end of 12-month treatment period
- Incidence of severe hypersensitivity and anaphylactic reactions — Baseline to end of 12-month treatment period
Trial sites (63)
| Facility | City | Region | Status |
|---|---|---|---|
| Intermountain - Primary Children's Hospital | Salt Lake City | Utah | Recruiting |
| Arbesu Hematología | Mendoza | Argentina | Recruiting |
| Murdoch Children's Research Institute | Parkville | Australia | Recruiting |
| Medizinische Universität Wien | Vienna | State of Vienna | Recruiting |
| Centro Estadual de Hemoterapia e Hematologia Marcos Daniel Santos - HEMOES | Maruípe | Vitória | Active Not Recruiting |
| Stollery Children's Hospital | Edmonton | Alberta | Active Not Recruiting |
| Hamilton Health Sciences - McMaster University/McMaster Children's Hospital | Hamilton | Ontario | Active Not Recruiting |
| Beijing Children's hospital, Capital Medical University | Beijing | Beijing Municipality | Recruiting |
| Southern Medical University Nanfang Hospital | Guangzhou | Guangdong | Active Not Recruiting |
| The Affiliated Hospital of Guizhou Medical University | Guiyang | Guizhou | Recruiting |
| Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology | Wuhan | Hubei | Recruiting |
| Tongji Hospital of Tongji Medical College of HUST/Pediatric Hematology Department Pharmacy | Wuhan | Hubei | Recruiting |
| Jiangxi Provincial People's Hospital | Nanchang | Jiangxi | Active Not Recruiting |
| Institute of hematology&blood disease hospital | Tianjin | Tianjin Municipality | Recruiting |
| Detska nemocnice FN Brno | Brno | Brno-město | Recruiting |
| Fakultni nemocnice Brno | Brno | Czechia | Recruiting |
| Fakultni Nemocnice Motol a Homolka | Prague | Czechia | Recruiting |
| Rigshospitalet | Copenhagen | Capital Region | Recruiting |
| Aarhus Universitetshospital, Skejby | Aarhus N | Denmark | Recruiting |
| Aarhus University | Aarhus N | Denmark | Recruiting |
| Charité Campus Virchow-Klinikum | Berlin | Germany | Recruiting |
| Nirmal Hospital Pvt Ltd. | Surat | Gujarat | Recruiting |
| K. J. Somaiya Hospital and Research Centre, Somaiya Ayurvihar Complex | Mumbai | Maharashtra | Recruiting |
| Nil Ratan Sircar Medical College and Hospital | Kolkata | West Bengal | Recruiting |
| Sheba Medical Center | Ramat Gan | Central District | Recruiting |
| IRCCS Istituto Giannina Gaslini | Genoa | Liguria | Recruiting |
| Istituto Clinico Humanitas - Humanitas Mirasole SPA | Rozzano | Milan | Recruiting |
| Ospedale Pediatrico Bambino Gesù IRCCS | Rome | ROMA | Recruiting |
| Azienda Ospedaliero Universitaria di Parma | Parma | Italy | Recruiting |
| Azienda Ospedaliero-Universitaria Città della Salute e della Scienza di Torino | Torino | Italy | Recruiting |
| Hyogo prefectural Kobe Children's Hospital | Kobe | Hyōgo | Recruiting |
| Nagano Children's Hospital | Azumino | Nagano | Active Not Recruiting |
| Nara Medical University Hospital | Kashihara | Nara | Recruiting |
| Saitama Prefectural Children's Medical Center | Saitama-shi | Saitama | Recruiting |
| Saga University Hospital | Saga | Japan | Active Not Recruiting |
| Arké SMO S.A de C.V | Veracruz | Mexico | Recruiting |
| King Fahad Specialist Hospital | Dammam | Saudi Arabia | Terminated |
| Detska fakultna nemocnica Kosice | Košice | Slovakia | Recruiting |
| Univerzitna nemocnica Martin | Martin | Slovakia | Recruiting |
| Worthwhile Clinical Trials | Benoni | Gauteng | Active Not Recruiting |
+ 23 more sites — see the full list on the official registry below.
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This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.
View NCT05611801 on ClinicalTrials.gov ↗ ← All trials in Italy