Fitusiran (SAR439774)

DRUG2 trials

Sponsors

Genzyme, a Sanofi Company, Sanofi

Conditions

Hemophilia AHemophilia B

Phase 1

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

CompletedNCT02554773

Genzyme, a Sanofi CompanyHemophilia A, Hemophilia B

Start: 2015-09-18End: 2023-03-21Updated: 2024-06-05

Phase 4

A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab

RecruitingNCT06145373

SanofiHemophilia A

Start: 2024-03-01End: 2030-01-30Target: 20Updated: 2026-06-02

Related Papers

Evaluating the safety and tolerability of switching from emicizumab to fitusiran prophylaxis in adult males with severe hemophilia A, with or without inhibitors: SWITCH study, a trial in progress

Blood2025-11-03

Experience with minor surgeries in people with hemophilia A or B with and without inhibitors receiving fitusiran

Blood2025-11-03

Long-term safety and efficacy of fitusiran prophylaxis, and perioperative management, in people with hemophilia A or B

Blood Advances2024-12-0614 citations

Fitusiran Population Pharmacokinetic and Pharmacodynamic Modeling Utilizing Phase 3 Clinical Trial Data to Confirm Doses Tested in Phase 3 Trials to Support an Antithrombin-Based Dose Regimen

Blood2023-11-02

Sustained Improvement in Health-Related Quality of Life in Patients with Hemophilia A with or without Inhibitors Treated with Fitusiran Prophylaxis

Blood2021-11-051 citations

Long-Term Durability, Safety and Efficacy of Fitusiran Prophylaxis in People with Hemophilia a or B, with or without Inhibitors - Results from the Phase II Study

Blood2020-11-058 citations

Longitudinal Assessment of Thrombin Generation in Patients with Hemophilia Receiving Fitusiran Prophylaxis: Phase II Study Results

Blood2020-11-043 citations

Fitusiran Treatment Impacts on Health-Related Quality of Life in Subjects with Hemophilia A and B with Inhibitors Assessed with the Haemophilia Quality of Life Questionnaire for Adults (Haem-A-QoL)

Blood2020-11-041 citations

Perioperative Management in Patients with Hemophilia Receiving Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia

Blood2017-12-0711 citations

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients without Inhibitors

Blood2016-12-0224 citations

Fitusiran, an Investigational RNAi Therapeutic Targeting Antithrombin for the Treatment of Hemophilia: Updated Results from a Phase 1 and Phase 1/2 Extension Study in Patients with Inhibitors

Blood2016-12-0218 citations