Trial intelligence / Profile preview

Fitusiran Prophylaxis After Switching From Emicizumab in Severe Hemophilia A

NCT06145373Phase 4

Key facts

Status: Recruiting
Start: Mar 2024
Primary completion: May 2028
Countries: 2
Sponsor: Sanofi
Modality: Conjugated siRNA, Chemically Modified siRNA

Drugs: fitusiran
Conditions: hemophilia A

Overview

Phase 4, open-label, single-arm trial of fitusiran in hemophilia A, sponsored by Sanofi. Primary endpoint: Number of participants with Adverse events. Started Mar 2024; status: recruiting.

Design

1 arm

Study design: Non-randomizedOpen-labelSingle group

fitusiran
Participants receive pre-fitusiran treatment (IV CFCs or BPAs) followed by SC fitusiran prophylaxis Q2M or QM for up to 18 months.

Who it enrols

Male participants aged >=12 years with severe hemophilia A (with or without inhibitors) currently receiving emicizumab prophylaxis.

Endpoints

Primary

Number of participants with Adverse eventsDay 1 to Month 4

Secondary

Peak thrombin generationMonth -2 to Day 1
Antithrombin levelsMonth -2 to Day 1
Emicizumab concentrations in plasmaUp to Month 4
Number of participants with Adverse eventsDay 1 to Month 18
Treatment Satisfaction Questionnaire for Medication domain scoresMonth -2 to Month 19
Participants' treatment preferencesMonth 12
Change in adult participants' pain intensityMonth -2 to Month 19
Change in adolescent participants' pain intensityMonth -2 to Month 19

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