RecruitingPHASE3INTERVENTIONAL

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

This study is looking into a new medicine called marstacimab for young people aged 1 to 17 who have haemophilia A or B. Haemophilia is a condition where blood doesn't clot properly, leading to bleeding. The main goal is to find out if marstacimab is safe and helps prevent these bleeding episodes. Participants will receive marstacimab as a weekly injection under the skin, often at home, for 12 months. Researchers will compare their bleeding experienced during the study with their past health records to see if the medicine makes a difference. The study involves regular check-ups and phone calls over about 14 months.

At a glance

Status

Recruiting

Phase

PHASE3

Sponsor

Pfizer

Enrolment target

100

Start

09 Dec 2022

Estimated completion

10 Sep 2028

Hemophilia A Hemophilia B

What is this study about?

Haemophilia A and B are conditions where your blood doesn't clot as it should, which can lead to bleeding that is hard to stop. This often means facing painful and sometimes serious bleeding episodes. Doctors and researchers are always looking for new treatments that can make life easier and safer for people with haemophilia, especially children and teenagers.

This study is focusing on a new medicine called marstacimab. We want to understand if this medicine is safe and if it can help prevent bleeding in young people (aged 1 to 17 years old) who have haemophilia A or haemophilia B. Participants will receive marstacimab as a weekly injection under the skin, similar to a regular shot you might get at the doctor's office, but you might be able to do this at home after the first dose.

The study will follow participants for about 14 months in total. During this time, the research team will keep a close eye on their health and any bleeding episodes. They will compare these experiences to how the participants were doing before the study, to see if marstacimab helps to reduce the frequency and severity of bleeding events.

Key takeaways

New medicine (marstacimab) for haemophilia A or B.
For boys and young men aged 1 to 17.
Aims to reduce bleeding episodes.
Weekly injection, potentially at home.
Study lasts about 14 months with clinic visits and calls.
Focuses on safety and effectiveness compared to past health.

Who may be eligible?

This study is open to boys and young men aged between 1 and 17 years old. They need to have a confirmed diagnosis of severe haemophilia A or moderate to severe haemophilia B. It's important that they have good health records from the past year that show their previous treatments and any bleeding episodes.

For those without 'inhibitors' (substances that can make haemophilia treatment harder), they must have been on a stable, regular preventative treatment for at least a year. If they do have inhibitors, they need to have been on a specific type of treatment for bleeding for at least a year before joining the study.

There are also weight requirements for different age groups: teenagers (12-17) must weigh at least 25 kg, and children (6-11) must weigh at least 19 kg. The minimum weight for the youngest group (1-5) will be confirmed later.

Could this study suit you?

Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.

Are you a male between 1 and 17 years old?
Do you have severe haemophilia A or moderate to severe haemophilia B?
Do you have health records for the past year showing your treatments and bleeding?
If you don't have 'inhibitors', have you been on a stable, regular preventative treatment for at least a year?
If you have 'inhibitors', have you been on a specific bleeding treatment for at least a year?
Do you meet the weight requirements for your age group (e.g., 25 kg for 12-17 year olds)?

Answer every question to see your result.

What does participation involve?

If you decide to take part, the study will last about 14 months. This includes an initial screening period of about a month, followed by 12 months when you’ll be receiving the study medicine, and then a month of follow-up. You will receive the study medicine, marstacimab, once a week as an injection just under the skin. The first dose will be given by the study staff, but after that, you might be able to give the weekly injections at home if you prefer and are able to. Over the 14 months, you will need to visit the study clinic at least 10 times. For some of these visits, if allowed, you might be able to have them at home instead. There will also be 6 phone calls spread out every couple of months to check in on you.

Potential risks and benefits

Taking part in this study may offer potential benefits, such as receiving a new medicine that might help reduce bleeding episodes. However, as with any medicine, there could be side effects or risks that your study doctor will discuss with you. You will be closely monitored by the study team, and you are free to withdraw from the study at any time, for any reason, without it affecting your medical care.

Locations (34)

Some site locations are approximate. We're improving this — please verify with the trial team before travelling.

Arbesu Hematología
Verified postcode
Mendoza, Argentina· Recruiting
Murdoch Children's Research Institute
Verified postcode
Parkville, Australia· Recruiting
Medizinische Universität Wien
Verified postcode
Vienna, Austria· Recruiting
Hamilton Health Sciences - McMaster University Medical Centre
Verified postcode
Hamilton, Canada· Recruiting
Tongji Hospital of Tongji Medical College of Huazhong University of Science and Technology
Verified postcode
Wuhan, China· Recruiting
Tongji Hospital of Tongji Medical College of HUST/Pediatric Hematology Department Pharmacy
Verified postcode
Wuhan, China· Recruiting
Institute of hematology&blood disease hospital
Verified postcode
Tianjin, China· Recruiting
Detska nemocnice FN Brno
Verified postcode
Brno, Czechia· Recruiting
Motol University Hospital
Verified postcode
Prague, Czechia· Recruiting
Rigshospitalet
Verified postcode
Copenhagen, Denmark· Recruiting
Aarhus Universitetshospital, Skejby
Verified postcode
Aarhus N, Denmark· Recruiting
Charité Campus Virchow-Klinikum
Verified postcode
Berlin, Germany· Recruiting

Common questions

What is haemophilia?

Haemophilia is a condition where your blood doesn't clot properly, which can lead to longer or more frequent bleeding than usual.

What is marstacimab?

Marstacimab is the new medicine being tested in this study to see if it can help prevent bleeding in people with haemophilia.

How is the medicine given?

The medicine is given as an injection once a week, just under the skin. You might be able to do this at home after the first dose.

How long does the study last?

The study lasts for about 14 months in total, including screening, treatment, and follow-up.

Can girls or women join this study?

No, this study is only recruiting male participants.

How to find out more

Pfizer CT.gov Call Center

clinicaltrials.gov_inquiries@pfizer.com 1-800-718-1021 Official trial record

Always speak to your GP or specialist before deciding to take part in a study.