Not recruitingRare disease

Study of Long-term Safety and Efficacy of Fitusiran in Patients with Hemophilia A or B with or without Inhibitory Antibodies to Factor VIII or IX

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This clinical trial is focused on studying hemophilia A and B, which are bleeding disorders where the blood doesn't clot properly. The therapy being investigated is called fitusiran, a medication designed to help control bleeding in people with these conditions. The aim of the study is to observe the long-term safety and how well fitusiran is tolerated by patients.

Participants in the study will receive fitusiran as a solution for injection. It is important to note that some patients involved might also have inhibitors to clotting factors, which means their bodies might reject certain treatments. In these cases, alternative treatments such as Factor VIII Inhibitor Bypassing Activity, Antithrombin III Human, Eptacog Alfa (Activated), Simoctocog Alfa, and Nonacog Alfa may be used. These are all different therapies aimed at helping the blood to clot more effectively.

The trial is designed to track the number of patients who experience any new health problems during the treatment, known as treatment emergent adverse events. It will also look at how often bleeding occurs, how this affects the joints, and the overall quality of life for those receiving fitusiran over a long period. Some participants might be given a placebo for comparison. The information gathered from this study will help better understand the long-term effects and safety of fitusiran in managing hemophilia.

The research process

The trial runs in 6 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Joining the study
Upon joining the study, the participant must have completed a previous Phase 3 clinical trial involving fitusiran.
The participant must be at least 12 years old and male, with a diagnosis of severe hemophilia A or hemophilia B.
Step 2
Treatment administration
The study involves the administration of fitusiran, which is given as a subcutaneous injection. This means the medication is injected under the skin.
Other medications may include factor viii inhibitor bypassing activity, antithrombin iii, coagulation factor viia, coagulation factor viii, and coagulation factor ix, which are administered intravenously (directly into a vein).
Step 3
Monitoring and evaluation
The main objective of the study is to assess the long-term safety and tolerability of fitusiran.
Participants will be monitored for any treatment emergent adverse events (TEAEs), which are any side effects that occur after starting the treatment.
Step 4
Assessment of bleeding rates
The study will evaluate the annualized bleeding rate during the treatment period, which includes the frequency of bleeding episodes over a year.
This assessment also includes the annualized spontaneous bleeding rate and joint bleeding rate.
Step 5
Quality of life evaluation
For participants aged 17 and older, changes in physical health scores will be measured using the haemophilia quality of life questionnaire for adults (HaemAQoL).
Step 6
Study duration
The study is expected to continue until June 18, 2026.
Participants will be involved in the study for the duration of the treatment period, with regular assessments and monitoring.

Who can join the trial?

4 criteria

The participant must be at least 12 years old at the time of signing the consent form.
The participant must have severe Hemophilia A or Hemophilia B and have completed a Phase 3 fitusiran clinical trial. Hemophilia is a condition where the blood doesn't clot properly, leading to excessive bleeding.
The participant must be male.
The participant must be able to give signed informed consent, which means they understand and agree to follow the study's rules and requirements. If the participant is not legally an adult, their legally authorized representative must also sign the consent form.

Who cannot join the trial?

3 criteria

Individuals who do not have Hemophilia A or Hemophilia B cannot participate. Hemophilia A and B are conditions where the blood does not clot properly, leading to excessive bleeding.
Females are not eligible to participate in this study.
Individuals outside the specified age ranges are not eligible. The age ranges include children, teenagers, and adults.

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

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Azienda Ospedaliera di Padova
Padua, Italy
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Centre Hospitalier Lyon Sud
Pierre Benite, France
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Fondazione IRCCS Ca Granda Ospedale Maggiore Policlinico
Milan, Italy

Trial locations

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Investigated drugs

Fitusiran is a medication being studied for its long-term safety and effectiveness in patients with Hemophilia A or B. Hemophilia is a condition where blood does not clot properly, leading to excessive bleeding. Fitusiran works by reducing the levels of a protein in the liver that helps control bleeding, potentially improving the blood's ability to clot. This study includes patients with or without inhibitory antibodies to Factor VIII or IX, which are proteins involved in the clotting process.

What is already known about the treatment

Fitusiran – Fitusiran is administered as a subcutaneous injection, meaning it is injected under the skin. It is currently being studied in clinical trials for its long-term safety and effectiveness in treating Hemophilia A and B, including cases with inhibitory antibodies to Factor VIII or IX. The medication works by targeting a specific molecule in the liver to reduce the production of antithrombin, which helps increase the generation of thrombin, a key protein in blood clotting. Fitusiran is classified as an RNA interference therapeutic, a type of medication that uses small RNA molecules to interfere with the expression of specific genes.

Investigated diseases

Hemophilia A
Hemophilia A is a genetic disorder where the blood does not clot properly due to a deficiency of clotting factor VIII. This condition leads to prolonged bleeding after injuries, surgeries, or even spontaneously without any apparent cause. Individuals with Hemophilia A may experience frequent nosebleeds, easy bruising, and joint bleeds, which can cause pain and swelling. Over time, repeated joint bleeds can lead to joint damage and mobility issues. The severity of symptoms can vary, with some individuals experiencing mild symptoms and others having more severe bleeding episodes. Hemophilia A is typically diagnosed in childhood, often after a significant bleeding event.
Hemophilia B
Hemophilia B is a genetic bleeding disorder caused by a deficiency of clotting factor IX. Similar to Hemophilia A, this condition results in the blood's inability to clot properly, leading to prolonged bleeding episodes. Individuals with Hemophilia B may experience spontaneous bleeding, especially into joints and muscles, which can cause pain and swelling. Over time, repeated bleeding into joints can lead to chronic joint damage and reduced mobility. The severity of Hemophilia B can range from mild to severe, depending on the level of factor IX activity in the blood. This condition is often identified in early childhood following unusual bleeding patterns.