Study on Marstacimab for Children 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 effectiveness and safety of a treatment called Marstacimab for children and teenagers with a condition known as Hemophilia. Hemophilia is a rare bleeding disorder where the blood doesn’t clot properly, leading to excessive bleeding. The study includes participants with two types of hemophilia: Hemophilia A, which involves a deficiency in a protein called factor VIII, and Hemophilia B, which involves a deficiency in factor IX. The trial is open to participants under 18 years of age, with severe forms of these conditions, whether or not they have developed inhibitors, which are antibodies that can interfere with treatment.

The purpose of the study is to compare the new treatment, Marstacimab, with the standard treatment that participants have been receiving over the past year. Marstacimab is administered as a solution for injection using a pre-filled pen, which is a device that allows the medication to be injected under the skin. The study will last for about 48 weeks, during which participants will receive regular doses of Marstacimab. The trial aims to see if Marstacimab can reduce the number of bleeding episodes and improve the overall health of the joints, which are often affected by bleeding in people with hemophilia.

Throughout the study, the safety of Marstacimab will be closely monitored, including any side effects or reactions at the injection site. The trial will also assess the impact of the treatment on the participants’ quality of life, using questionnaires designed for different age groups. This research is important for understanding how well Marstacimab works in young people with hemophilia and whether it can become a new option for managing this condition.

1 initial visit

Upon joining the study, an initial visit is scheduled. During this visit, eligibility is confirmed based on age, weight, and medical history related to hemophilia.

A detailed review of past treatment records and bleeding episodes is conducted to ensure all criteria are met.

2 baseline assessment

A baseline assessment is performed to evaluate current health status. This includes physical examinations and laboratory tests.

The assessment helps establish a starting point for measuring the effects of the study medication.

3 medication administration

The study involves the administration of marstacimab, a solution for injection provided in a pre-filled syringe.

The medication is administered subcutaneously (under the skin) at a frequency determined by the study protocol.

4 regular follow-up visits

Regular follow-up visits are scheduled to monitor health and response to the medication.

These visits include physical examinations, laboratory tests, and discussions about any side effects or concerns.

5 end of study evaluation

At the end of the study period, a final evaluation is conducted to assess the overall impact of the medication.

This includes a comprehensive review of health changes, bleeding episodes, and any side effects experienced during the study.

Who Can Join the Study?

  • Participants must be male.
  • Participants must be between 1 to 18 years old.
  • Participants must have a minimum body weight:
    • For ages 12 to <18 years: at least 25 kg (about 55 pounds).
    • For ages 6 to <12 years: at least 19 kg (about 42 pounds).
    • For ages 1 to <6 years: minimum weight will be determined based on previous data.
  • Participants must have a diagnosis of severe hemophilia A or moderately severe to severe hemophilia B. This means their blood clotting factor levels are very low.
  • Participants must have at least 1 year of medical records showing their treatment and bleeding episodes.
  • For the Non-Inhibitor Cohort:
    • No detectable inhibitors against clotting factors.
    • No history of high-level inhibitors in the past 5 years.
    • No low-level inhibitors that do not respond to treatment in the past 5 years.
    • If they had immune tolerance therapy (ITI), it must have been completed successfully at least 5 years ago.
    • At least 50 documented treatment days with clotting factor products.
    • Must be on a stable routine treatment to prevent bleeding, with at least 80% compliance over the past year.
  • For the Inhibitor Cohort:
    • Current high-level inhibitors or low-level inhibitors that do not respond to treatment.
    • Participants with a history of inhibitors may be considered on a case-by-case basis.
    • For hemophilia A: at least 12 bleeding episodes in the past year requiring treatment.
    • For hemophilia B: at least 8 bleeding episodes in the past year requiring treatment.
    • Must be on an on-demand treatment regimen, with documented bleeding episodes over the past year.
    • Participants on routine treatment to prevent bleeding must have at least 80% compliance over the past year.

Who Cannot Join the Study?

  • Participants must not have any other serious medical conditions that could interfere with the study.
  • Participants must not have a history of allergic reactions to the study medication or similar medications.
  • Participants must not be taking any other medications that could interfere with the study medication.
  • Participants must not have any bleeding disorders other than hemophilia.
  • Participants must not have any active infections that require treatment.
  • Participants must not have any liver disease or liver function problems.
  • Participants must not have any kidney disease or kidney function problems.
  • Participants must not have any heart disease or heart function problems.
  • Participants must not have any history of drug or alcohol abuse.
  • Participants must not have any mental health conditions that could interfere with the study.
  • Participants must not have participated in another clinical trial within the last 30 days.
  • Participants must not have received any blood products within the last 30 days.
  • Participants must not have any planned surgeries during the study period.

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
University Hospital Bratislava Bratislava Slovakia
Detska Fakultna Nemocnica Kosice Kosice Slovakia
Fakultni Nemocnice Brno Brno Czechia
Region Midtjylland Aarhus Denmark
Rigshospitalet Copenhagen Denmark
IRCCS Istituto Giannina Gaslini Genoa Italy
Deutsches Herzzentrum Berlin Berlin Germany
Univerzitna Nemocnica Martin Martin Slovakia
Ospedale Pediatrico Bambino Gesu’ Rome Italy
Amevshv Oelpdhewcha Uiskwavokhotz Cfvtassdecby Dljnf Sgmfsu E Diaoz Shfulam Dr Ttmvwk Turin Italy
Fxtouwkz ntwhwqhbi Moptc a Hqlgynp Prague Czechia
Atipxqa Ouakzfnusci Ubostbgieaqun Pudcf Parma Italy
Fttakbunh Prlw Lc Iajdckgyapzxu Bpvaqptpv Dca Hwcuuwmx Uwcccetxzuucs Lj Pau Madrid Spain

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

Trial status

Country Status Recruitment Start
Austria Austria
Recruiting
21.04.2023
Czechia Czechia
Recruiting
21.04.2023
Denmark Denmark
Recruiting
21.04.2023
France France
Not recruiting
21.04.2023
Germany Germany
Recruiting
21.04.2023
Italy Italy
Recruiting
21.04.2023
Slovakia Slovakia
Recruiting
21.04.2023
Spain Spain
Recruiting
21.04.2023

Trial locations

Investigated drugs:

Marstacimab is a medication being studied for its potential to help people with severe hemophilia A or moderately severe to severe hemophilia B. Hemophilia is a condition where the blood does not clot properly, leading to excessive bleeding. Marstacimab is designed to be used as a regular treatment to prevent bleeding episodes. It works by helping the blood to clot more effectively, which can reduce the frequency and severity of bleeding events. This study is looking at how well marstacimab works and how safe it is for children under 18 years old who have hemophilia, with or without inhibitors, which are substances that can interfere with treatment.

Hemophilia – Hemophilia is a genetic disorder that affects the blood’s ability to clot properly. It is caused by a deficiency in one of the clotting factors, typically factor VIII (Hemophilia A) or factor IX (Hemophilia B). This deficiency leads to prolonged bleeding after injuries, surgeries, or even spontaneously without any apparent cause. Over time, repeated bleeding episodes, especially into joints and muscles, can lead to joint damage and pain. The severity of hemophilia varies, with severe cases experiencing frequent bleeding episodes and milder cases having fewer issues. The condition is usually inherited and primarily affects males, though females can be carriers and occasionally have symptoms.

Trial ID:
2022-500495-65-00
Protocol code:
B7841008
NCT ID:
NCT05611801
Trial Phase:
Therapeutic confirmatory (Phase III)

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