Fitusiran for Hemophilia
(ATLAS-OLE Trial)
What You Need to Know Before You Apply
What is the purpose of this trial?
This trial examines fitusiran, a treatment designed to improve life for individuals with severe hemophilia A or B. It evaluates the long-term safety of fitusiran and its effectiveness in reducing bleeding episodes, including joint bleeds. The trial also assesses whether the treatment enhances participants' quality of life. It is suitable for males aged 12 and older with severe hemophilia who have completed a previous fitusiran Phase 3 trial. As a Phase 3 trial, this study represents the final step before FDA approval, providing participants an opportunity to contribute to a potentially groundbreaking treatment.
Will I have to stop taking my current medications?
The trial does not specify if you need to stop taking your current medications, but it does exclude those using certain hemophilia treatments like factor concentrates or bypassing agents regularly. It's best to discuss your specific medications with the trial team.
Is there any evidence suggesting that fitusiran is likely to be safe for humans?
Research has shown that fitusiran appears promising in terms of safety for people with hemophilia. In earlier studies, fitusiran was administered as a monthly shot. The results demonstrated a significant reduction in bleeding episodes compared to standard treatments, suggesting that fitusiran effectively manages bleeding.
One study found that adjusting the target levels of antithrombin, a protein that helps control bleeding, enhanced fitusiran's safety while still preventing bleeding. This adjustment improved fitusiran's safety profile for patients.
Another study examined the long-term safety of fitusiran and found it to be well tolerated. While potential risks exist with any treatment, these studies provide encouraging signs that fitusiran is generally safe for people with hemophilia.12345Why do researchers think this study treatment might be promising for hemophilia?
Unlike the standard of care for hemophilia, which often involves regular intravenous infusions of clotting factor concentrates, Fitusiran is unique because it is administered as a subcutaneous injection. This treatment offers a new mechanism of action by targeting antithrombin, a protein that normally inhibits blood clotting, to enhance the body’s ability to form clots. Researchers are excited about Fitusiran because it has the potential to simplify treatment regimens with once-monthly or every-other-month dosing, potentially improving patients' quality of life and adherence to therapy.
What evidence suggests that fitusiran might be an effective treatment for hemophilia?
Research has shown that fitusiran reduces bleeding episodes in people with hemophilia A or B. Studies have found that fitusiran lowers antithrombin levels, a protein that controls blood clotting, which then increases thrombin production. This process helps blood clot more normally. Data indicates that people treated with fitusiran experienced fewer bleeds overall, including spontaneous and joint bleeds. Various studies have tested the treatment, showing positive effects on reducing bleeding frequency and potentially improving quality of life for those with hemophilia. Participants in this trial will receive fitusiran as a subcutaneous injection once monthly or every other month.26789
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 or older with severe hemophilia A or B who have finished a Phase 3 fitusiran study. They must be able to consent and follow the study rules. It's not for those on certain other treatments, with liver issues, history of blood clots, recent surgery, or in gene therapy trials.Inclusion Criteria
Exclusion Criteria
Timeline for a Trial Participant
Screening
Participants are screened for eligibility to participate in the trial
Treatment
Participants receive fitusiran as a subcutaneous injection once monthly or every other month
Follow-up
Participants are monitored for safety and effectiveness after treatment
Open-label extension
Participants may continue receiving fitusiran until it becomes commercially available
What Are the Treatments Tested in This Trial?
Interventions
- Fitusiran
Trial Overview
The trial tests the long-term safety and effectiveness of Fitusiran in managing bleeding episodes in hemophilia patients. It looks at how often bleeding occurs and its impact on quality of life over time.
How Is the Trial Designed?
1
Treatment groups
Experimental Treatment
Participants will be administered fitusiran as a subcutaneous injection once monthly or every other month for up to 48 months post initiation of modified IMP dose/frequency or until fitusiran becomes commercially available, whichever comes first.
Find a Clinic Near You
Who Is Running the Clinical Trial?
Genzyme, a Sanofi Company
Lead Sponsor
David Meeker
Genzyme, a Sanofi Company
Chief Executive Officer since 2011
MD from the University of Vermont Medical School, Advanced Management Program at Harvard Business School
Jean-Paul Kress
Genzyme, a Sanofi Company
Chief Medical Officer since 2015
MD from Faculte Necker-Enfants Malades, Paris
Published Research Related to This Trial
Citations
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 ...
2.
ashpublications.org
ashpublications.org/bloodadvances/article/9/5/1147/534349/Long-term-safety-and-efficacy-of-fitusiranLong-term safety and efficacy of fitusiran prophylaxis, and ...
The results of this long-term study indicate that fitusiran prophylaxis was effective at reducing AT and restoring sufficient TG to achieve hemostasis and ...
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- ...
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 ...
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 inhibitors ...
6.
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) ...
NCT03754790 | Long-term Safety and Efficacy Study of ...
Long-term Safety and Efficacy Study of Fitusiran in Patients With Hemophilia A or B, With or Without Inhibitory Antibodies to Factor VIII or IX (ATLAS-OLE).
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.
Press Release: Two fitusiran Phase 3 studies published in ...
The ATLAS A/B study published in The Lancet Haematology showed 51% of participants without inhibitors (40 out of 79) who received fitusiran 80mg ...
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