What side effects are associated with this type of intervention?

Common treatments for hemophilia, particularly those involving small interfering RNA (siRNA) like Fitusiran, can have side effects such as thrombosis due to enhanced thrombin generation. Other advanced therapies, including recombinant factors and bypassing agents, may cause immune responses leading to inhibitor development, allergic reactions, and injection site reactions. Additionally, treatments like emicizumab can lead to thrombotic microangiopathy and thromboembolism in rare cases. It is crucial to monitor patients closely for these potential adverse effects.

What prior FDA approvals exist?

Fitusiran is a novel treatment for Hemophilia that uses small interfering RNA (siRNA) to target antithrombin, thereby enhancing thrombin generation and improving hemostasis. While there are no other siRNA treatments currently approved by the FDA specifically for Hemophilia, several other therapies have been approved. These include recombinant factor VIII and IX products, such as Eloctate (rFVIII-Fc) and Idelvion (rFIX-FP), which are used for prophylaxis and treatment of bleeding episodes. Additionally, Emicizumab, a bispecific monoclonal antibody, has been approved for Hemophilia A patients with and without inhibitors, offering a different mechanism to improve hemostasis.

What other types of research exist?

Promising alternatives to Fitusiran for Hemophilia patients include: 1) Emicizumab, a bispecific monoclonal antibody that mimics the function of factor VIII, showing reduced annualized bleeding rates in clinical trials. 2) Gene therapies like valoctocogene roxaparvovec for Hemophilia A and etranacogene dezaparvovec for Hemophilia B, which aim to provide long-term correction by introducing functional copies of the defective gene. 3) Other siRNA therapies targeting different pathways involved in coagulation are also under investigation.

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RNAi Therapeutics

Fitusiran for Hemophilia (ATLAS-NEO Trial)

Phase 3

Waitlist Available

Research Sponsored by Sanofi

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Timeline

Screening 3 weeks

Treatment Varies

Follow Up day 1 (d1) to day 505, and from d1 to day 1009, and during soc from day-168 to d-1

Awards & highlights

No Placebo-Only Group

Pivotal Trial

Summary

This trial tests fitusiran, a medication that helps reduce bleeding in male adults and adolescents with hemophilia A or B. It works by lowering antithrombin levels to improve blood clotting.

Who is the study for?

This trial is for male teens and adults with severe Hemophilia A or B, who've had at least 4 bleeding episodes in the last 6 months. They must be willing to follow study rules and give consent. Those with liver disease, thrombophilic disorders, certain viral infections, low CD4 counts if HIV positive, poor kidney function, other bleeding disorders besides hemophilia or a history of blood clots can't join.

What is being tested?

The trial tests Fitusiran injections under the skin against standard treatments (clotting factors or bypassing agents) for preventing bleeds in Hemophilia patients. It's an open-label study where participants switch from their current treatment to Fitusiran for about three years after a six-month observation period on their usual care.

What are the potential side effects?

Fitusiran may cause injection site reactions and has potential risks like developing blood clots due to its effect on clotting proteins. Standard treatments' side effects vary but include allergic reactions and complications related to long-term use.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ day 1 (d1) to day 505, and from d1 to day 1009, and during soc from day-168 to d-1

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~day 1 (d1) to day 505, and from d1 to day 1009, and during soc from day-168 to d-1

This trial's timeline: 3 weeks for screening, Varies for treatment, and day 1 (d1) to day 505, and from d1 to day 1009, and during soc from day-168 to d-1 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.

Primary study objectives

Annualized bleeding rate (ABR) in the fitusiran primary efficacy period

Secondary study objectives

Annualized bleeding rate (ABR) in the fitusiran 18-month efficacy period

Annualized bleeding rate (ABR) in the fitusiran 36-month treatment period

Annualized bleeding rate (ABR) while on fitusiran prophylaxis in the fitusiran primary efficacy period and ABR while on on-demand standard of care (SOC) in the SOC period

+6 more

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Pivotal Trial

The final step before approval, pivotal trials feature drugs that have already shown basic safety & efficacy.

Trial Design

1Treatment groups

Experimental Treatment

Group I: All participantsExperimental Treatment3 Interventions

In standard of care (SOC) period, participants will receive on-demand or prophylactic treatment with clotting factor concentrates (CFCs) or bypassing agents (BPAs) for 6 months (from Day -168 to Day -1). In fitusiran treatment period, participants will receive subcutaneous fitusiran prophylaxis once every other month (Q2M) or once monthly (QM) for 36 months (from Day 1 to Day 1009). In case of bleeding events participants will receive IV CFCs or BPAs. Participants may receive Antithrombin concentrate (ATIIIC) as rescue medicine. .

Treatment

First Studied

Drug Approval Stage

How many patients have taken this drug

Fitusiran

2018

Completed Phase 3

~80

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Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Common treatments for hemophilia include factor replacement therapy, bypassing agents, and newer therapies like small interfering RNA (siRNA) treatments. Factor replacement therapy involves infusing the missing clotting factors (Factor VIII for Hemophilia A and Factor IX for Hemophilia B) directly into the bloodstream to prevent or control bleeding. Bypassing agents, such as recombinant activated factor VII, are used in patients with inhibitors to traditional factor replacement, helping to form a clot by bypassing the need for the missing factor. Fitusiran, an siRNA treatment, targets antithrombin to reduce its levels, thereby enhancing thrombin generation and improving hemostasis. This is particularly important for hemophilia patients as it offers a novel approach to managing bleeding episodes, especially for those with inhibitors to standard treatments, by promoting more effective clot formation.

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