Study on Long-Term Safety and Effects of Marstacimab for Patients with Severe Hemophilia A or B, With or Without Inhibitors

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What is this study about?

This clinical trial is focused on studying the long-term safety and effectiveness of a treatment called Marstacimab for people with a condition known as hemophilia. Hemophilia is a rare bleeding disorder where blood doesn’t clot properly, leading to excessive bleeding. The study includes individuals with severe hemophilia A or moderately severe to severe hemophilia B, with or without inhibitors. Inhibitors are substances that can interfere with the treatment of hemophilia, making it more challenging to manage the condition.

The purpose of this study is to evaluate how safe and tolerable Marstacimab is when used over a long period. Participants will receive the treatment as a solution for injection, either in a pre-filled pen or syringe, which is administered under the skin. The study will monitor participants for any side effects, including reactions at the injection site, changes in vital signs, and any significant changes in laboratory test results. The study will also track the number of bleeding episodes and any changes in joint health over time.

Participants in this study will be observed for several years to gather comprehensive data on the treatment’s impact. The study aims to provide valuable insights into the long-term management of hemophilia using Marstacimab, helping to improve the quality of life for those affected by this condition. The study is open-label, meaning both the researchers and participants know what treatment is being administered, and it is an extension of previous studies, allowing for continued observation and data collection.

1 joining the study

Upon joining the study, you will be asked to provide written or electronically signed consent. This ensures that you understand the study and agree to participate. If you are underage, your legal guardian will also need to provide consent.

2 initial assessment

An initial assessment will be conducted to confirm your eligibility. This may include reviewing your medical history and conducting some laboratory tests.

3 treatment administration

You will receive the medication marstacimab as a solution for injection. This will be administered subcutaneously, which means it is injected under the skin. The specific dosage and frequency will be determined by the study team based on your individual needs.

4 regular follow-up visits

You will have regular follow-up visits scheduled throughout the study. During these visits, the study team will monitor your health, check for any side effects, and assess the effectiveness of the treatment.

5 reporting side effects

It is important to report any side effects or unusual symptoms you experience during the study. This helps the study team ensure your safety and adjust the treatment if necessary.

6 completion of the study

At the end of the study, a final assessment will be conducted. This will include a review of your overall health and any changes observed during the study period.

Who Can Join the Study?

  • Participants must have hemophilia, which is a condition that affects the blood’s ability to clot.
  • Participants must be between 1 to 74 years old.
  • Participants must be male.
  • Participants must have been part of and successfully completed either Study B7841005 or Study B7841008 without needing to leave the study early.
  • Participants must have met the age and weight requirements in their previous study (either Study B7841005 or Study B7841008).
  • Participants must be willing and able to attend all scheduled visits, follow the treatment plan, and complete all required tests and procedures.
  • Participants or their legal guardians must provide written consent to participate in the study. This means they agree to join the study after being fully informed about what it involves.

Who Cannot Join the Study?

  • Patients who do not have severe hemophilia A or moderately severe to severe hemophilia B cannot participate. Hemophilia A and B are conditions where the blood does not clot properly.
  • Patients with Factor VIII (FVIII) activity of 1% or more cannot participate. FVIII is a protein that helps blood to clot.
  • Patients with Factor IX (FIX) activity of more than 2% cannot participate. FIX is another protein important for blood clotting.
  • Only male patients can participate. Female patients are not eligible.
  • Patients who are not within the age range of 1 to less than 75 years cannot participate.

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name City Country Status
IRCCS Humanitas Research Hospital Rozzano Italy
Medical University Of Vienna Vienna Austria
Hopital Necker Enfants Malades Paris France

Other Sites

Site Name City Country Status
IRCCS Istituto Giannina Gaslini Genoa Italy
Univerzitna Nemocnica Martin Martin Slovakia
Detska Fakultna Nemocnica Kosice Kosice Slovakia
KBC Zagreb Zagreb Croatia
Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico Milan Italy
Fakultni Nemocnice Brno Brno Czechia
Rigshospitalet Copenhagen Denmark
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Ajxkgdd Ooednfajjda Uxoknrtxipkmr Prodh Parma Italy
Akgueu Uaznmacptw Hcwqcyiu Aarhus Denmark
Afggmnm Otgrueomuft Uogtavhhmiusc Culxvwhpzfwt Dnesq Srzbah E Dijql Sttpttt Db Tdljeg Turin Italy
Ujpcdimjtu Dxpnl Sebbl Dy Rwle Lv Suofrvye Rome Italy
Fddkbbshf Plso Lo Iafwnmemkprlr Bwqbtzqfn Dod Hvzicldn Uollezihkdspu Lz Pzg Madrid Spain
Hiysofbz Vaci dwhkrtzw Barcelona Spain
Hzjxubgy Ujxihgqcqsnvt dg A Cojbys A Coruna Galicia Spain

Want to learn more about this study or check if you can participate? Contact us.

Trial status

Country Status Recruitment Start
Austria Austria
Not yet recruiting
16.11.2022
Croatia Croatia
Recruiting
16.11.2022
Czechia Czechia
Not yet recruiting
16.11.2022
Denmark Denmark
Recruiting
16.11.2022
France France
Recruiting
16.11.2022
Germany Germany
Not yet recruiting
16.11.2022
Italy Italy
Recruiting
16.11.2022
Slovakia Slovakia
Recruiting
16.11.2022
Spain Spain
Recruiting
16.11.2022

Trial locations

Investigated drugs:

Marstacimab is a medication being studied for its potential to help people with hemophilia, a condition where blood doesn’t clot properly. This medication is designed to be used as a long-term treatment to prevent bleeding episodes in people with severe hemophilia A or moderately severe to severe hemophilia B. It works by helping the blood to clot more effectively, which can reduce the risk of bleeding. The study is looking at how safe and well-tolerated marstacimab is when used over a long period, as well as how effective it is in preventing bleeding in people with hemophilia, whether or not they have developed inhibitors, which are substances that can make treatment less effective.

Hemophilia A – Hemophilia A is a genetic disorder caused by a deficiency of clotting factor VIII, which leads to prolonged bleeding. This condition primarily affects males and is characterized by spontaneous bleeding episodes, particularly into joints and muscles. Over time, repeated bleeding into joints can cause joint damage and pain. The severity of the disease varies, with severe cases having less than 1% of normal factor VIII activity. Individuals with this condition may experience frequent nosebleeds, easy bruising, and excessive bleeding after injuries or surgeries. The progression of the disease can lead to chronic joint problems and reduced mobility if not managed properly.

Hemophilia B – Hemophilia B is a genetic disorder resulting from a deficiency of clotting factor IX, leading to prolonged bleeding. Like Hemophilia A, it primarily affects males and is characterized by spontaneous bleeding episodes, especially into joints and muscles. The severity of Hemophilia B varies, with severe cases having less than 2% of normal factor IX activity. Repeated bleeding into joints can cause joint damage and chronic pain over time. Individuals with this condition may experience frequent nosebleeds, easy bruising, and excessive bleeding following injuries or surgeries. Without proper management, the disease can lead to chronic joint issues and decreased mobility.

Trial ID:
2022-500470-33-00
Protocol code:
B7841007
NCT ID:
NCT05145127
Trial Phase:
Therapeutic confirmatory (Phase III)

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