Fitusiran for Hemophilia A
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial evaluates the safety and tolerability of switching from emicizumab to fitusiran, an experimental treatment, for individuals with severe hemophilia A, a condition where blood doesn't clot properly. The researchers aim to assess fitusiran's effectiveness after discontinuing emicizumab, with a focus on the transition period. The study targets males aged 12 and older with severe hemophilia A who are currently receiving emicizumab treatments. Participants will receive fitusiran injections either once a month or every two months. Those managing severe hemophilia A and currently on emicizumab may qualify for this trial. As a Phase 4 trial, this study involves an FDA-approved treatment and seeks to understand how fitusiran benefits more patients.
Do I have to stop taking my current medications for the trial?
The trial requires participants to switch from emicizumab to fitusiran, so you will need to stop taking emicizumab. The protocol does not specify if you need to stop other medications, so it's best to discuss this with the trial team.
What is the safety track record for fitusiran?
Research shows that fitusiran is generally well-tolerated by people with hemophilia. Studies have demonstrated that it effectively reduces bleeding episodes over time. Regarding safety, a small risk of blood clots exists, but previous studies managed this well. The chance of blood clots occurring was low, about 0.82 per 100 patient-years.
Side effects, such as viral infections, occurred in about 29% of patients, but these were not always serious. Importantly, adjusting the fitusiran dose has improved its safety while still providing good protection against bleeding.
Overall, fitusiran has shown a positive safety record in people with hemophilia, supported by data from multiple studies over time.12345Why are researchers enthusiastic about this study treatment?
Researchers are excited about fitusiran for hemophilia A because it offers a unique approach compared to standard treatments, which typically involve regular intravenous infusions of clotting factor concentrates or bypassing agents. Fitusiran works by reducing the production of antithrombin, a protein that inhibits blood clotting, thereby enhancing the body’s ability to form clots naturally. This mechanism is different from the usual methods, which directly replace missing clotting factors. Additionally, fitusiran is administered subcutaneously, either once a month or every two months, which could significantly improve convenience and quality of life for patients compared to frequent intravenous treatments. Overall, this novel method could simplify treatment regimens and potentially improve outcomes for hemophilia A patients.
What is the effectiveness track record for fitusiran in treating hemophilia A?
Research has shown that fitusiran, which participants in this trial will receive, can effectively treat hemophilia. In earlier studies, fitusiran significantly reduced bleeding episodes in people with hemophilia A. It lowers a protein called antithrombin, helping the blood clot more easily. This results in fewer bleeds compared to traditional treatments used only as needed. Overall, fitusiran has helped people with hemophilia achieve better control over bleeding.23678
Who Is on the Research Team?
Clinical Sciences & Operations
Principal Investigator
Sanofi
Are You a Good Fit for This Trial?
This trial is for males aged ≥12 with severe hemophilia A, who were previously on emicizumab prophylaxis. They must have a certain level of inhibitors in their blood or historical evidence of such levels and be able to consent to the study's requirements.Inclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Pre-fitusiran Treatment
Participants receive on-demand or prophylactic treatment with intravenous clotting factor concentrates or bypassing agents
Fitusiran Treatment
Participants receive subcutaneous fitusiran prophylaxis once every 2 months or once monthly
Antithrombin Follow-up
Participants are monitored for antithrombin activity levels until they return to at least 60%
What Are the Treatments Tested in This Trial?
Interventions
- Fitusiran
Trial Overview
The study tests fitusiran as a preventive treatment for bleeds in participants after switching from emicizumab. It includes screening, transition period before starting fitusiran, an 18-month treatment phase with monthly visits, and a follow-up monitoring antithrombin activity.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
The pre-fitusiran treatment period is defined as the transition period up to the first fitusiran administration. Participants will receive on demand or prophylactic treatment with intravenous clotting factor concentrates (IV CFCs) or bypassing agents (BPAs) from Month-2 until Day 1. Fitusiran treatment period: Participants will receive subcutaneous (SC) fitusiran prophylaxis once every 2 months (Q2M) or once monthly (QM) from Day 1 until Month18. Participants may receive IV antithrombin concentrate (ATIIIC) upon investigator's judgement. AT FU period: Participants will be followed up until AT activity levels recover to at least 60% (per central laboratory). In case of bleeding events participants will receive IV CFCs or BPAs. Participants may receive IV ATIIIC upon investigator's judgement.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Sanofi
Lead Sponsor
Paul Hudson
Sanofi
Chief Executive Officer since 2019
Degree in Economics from Manchester Metropolitan University
Christopher Corsico
Sanofi
Chief Medical Officer
MD from Cornell University, MPH in Chronic Disease Epidemiology from Yale University
Citations
1.
ashpublications.org
ashpublications.org/blood/article/145/25/2966/535965/Safety-and-efficacy-of-a-fitusiran-antithrombinSafety and efficacy of a fitusiran antithrombin-based dose ...
In completed phase 3 trials, once-monthly 80 mg fitusiran prophylaxis significantly reduced annualized bleeding rate (ABR) vs on-demand CFC/bypassing agent (BPA) ...
Long-term safety and efficacy of fitusiran prophylaxis, and ...
Here, we present the results of a completed phase 2 open-label extension study, which evaluated the long-term safety and efficacy of fitusiran ...
Data from two Phase 3 studies demonstrating fitusiran ...
Data from two Phase 3 studies demonstrating fitusiran significantly reduced bleeds in people with hemophilia A or B, with or without ...
A Study of Fitusiran in Severe Hemophilia A and B Patients ...
Study had 2 main periods: 6-month factor/bypassing agent (BPA) prophylaxis period & 7-month fitusiran treatment period (1-month onset and 6-month fitusiran ...
Safety and efficacy of a fitusiran antithrombin-based dose ...
Fitusiran lowers AT to increase thrombin generation and restore hemostasis in people with hemophilia A/B with or without inhibitors. •. The AT- ...
Sanofi announces positive long-term efficacy and safety ...
Long-term exploratory data show prophylactic treatment with fitusiran provides a sustained reduction in annual bleed rates in moderate to severe ...
QFITLIA® (fitusiran) Safety | For US HCPs
Adverse reactions reported in ≥5% of patients treated with the AT-DR of Qfitlia (N=286)1. Adverse reaction, % of patients. Viral infection*, 29.
SAFETY AND EFFICACY OF THE FITUSIRAN REVISED ...
In this study, the fitusiran AT-DR targeting AT levels of 15%–35% improved the safety profile substantially versus the ODR and maintained bleed protection.
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