RECRUITING

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

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Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

This is an exploratory, single group, Phase 4, study to assess treatment with fitusiran prophylaxis after switching from emicizumab prophylaxis. This study aims to evaluate the safety and tolerability of switching to fitusiran after a transition period from the last dose of emicizumab. The study will be conducted in male participants with severe hemophilia A, with or without inhibitors, aged ≥12 years, who were previously receiving emicizumab prophylaxis. Study details include: * The study duration will be up to approximately 28 months: * There will be an approximately 2-month screening period. * There will be an approximately 2-month period before fitusiran treatment starts (pre-fitusiran treatment period) * The fitusiran treatment duration will be up to 18-months (fitusiran treatment period) * The antithrombin (AT) follow-up (FU) period will be approximately 6 months after the last dose of fitusiran (during which the AT activity level will be monitored at approximately monthly intervals following the final fitusiran dose until AT activity levels return to at least 60%). * The study site visits are scheduled at monthly/ every 2 months intervals of 28 days (4 weeks) / 56 days (8 weeks), respectively, during the fitusiran treatment period.

Official Title

An Open-label, Single-arm Treatment Study to Investigate the Safety and Tolerability of Switching From Emicizumab to Fitusiran Prophylaxis in Male Participants Aged ≥12 Years of Age With Severe Hemophilia A, With or Without Inhibitors

Quick Facts

Study Start:2024-03-01

Study Completion:2029-11-30

Study Type:Not specified

Phase:Not Applicable

Enrollment:Not specified

Status:RECRUITING

Study ID

NCT06145373

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:12 Years

Sexes Eligible for Study:MALE

Accepts Healthy Volunteers:No

Standard Ages:CHILD, ADULT, OLDER_ADULT

Inclusion Criteria	Exclusion Criteria
* Male participants must be ≥12 years of age inclusive, at the time of signing the informed consent * Diagnosis of severe congenital hemophilia A (FVIII \< 1%) as evidenced by a central laboratory measurement at screening or documented medical record evidence. * Inhibitor titer of ≥0.6 BU/mL at Screening, or * Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, or * Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of anamnestic response. * Participants who are currently on the full labeled dose of emicizumab prophylaxis, irrespective of inhibitor/non-inhibitor status. * Signed informed consent/assent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.	* Known coexisting bleeding disorders * History of antiphospholipid antibody syndrome. * History of arterial or venous thromboembolism, atrial fibrillation, significant valvular disease, myocardial infarction, angina, transient ischemic attack, or stroke. Participants who have experienced thrombosis associated with indwelling venous access may be enrolled. * Presence of clinically significant liver disease * Current or prior participation in a fitusiran trial * Current or prior participation in a gene therapy trial * AT activity \<60% at Screening, as determined by central laboratory measurement * Coexisting thrombophilic disorder - Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis * Presence of acute hepatitis, ie, hepatitis A, hepatitis E. * Presence of acute or chronic hepatitis B infection * Known to be HIV positive with CD4 count \<200 cells/μL. * Reduced renal function

Inclusion Criteria

Exclusion Criteria

* Male participants must be ≥12 years of age inclusive, at the time of signing the informed consent
* Diagnosis of severe congenital hemophilia A (FVIII \< 1%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
* Inhibitor titer of ≥0.6 BU/mL at Screening, or
* Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of 2 consecutive titers ≥0.6 BU/mL, or
* Inhibitor titer of \<0.6 BU/mL at Screening with medical record evidence of anamnestic response.
* Participants who are currently on the full labeled dose of emicizumab prophylaxis, irrespective of inhibitor/non-inhibitor status.
* Signed informed consent/assent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in this protocol.

* Known coexisting bleeding disorders
* History of antiphospholipid antibody syndrome.
* History of arterial or venous thromboembolism, atrial fibrillation, significant valvular disease, myocardial infarction, angina, transient ischemic attack, or stroke. Participants who have experienced thrombosis associated with indwelling venous access may be enrolled.
* Presence of clinically significant liver disease
* Current or prior participation in a fitusiran trial
* Current or prior participation in a gene therapy trial
* AT activity \<60% at Screening, as determined by central laboratory measurement
* Coexisting thrombophilic disorder - Hepatitis C virus antibody positive, except participants who have negative Hepatitis C viral load and no evidence of cirrhosis
* Presence of acute hepatitis, ie, hepatitis A, hepatitis E.
* Presence of acute or chronic hepatitis B infection
* Known to be HIV positive with CD4 count \<200 cells/μL.
* Reduced renal function

Contacts and Locations

Study Contact

Trial Transparency email recommended (Toll free for US & Canada)

CONTACT

800-633-1610

Contact-US@sanofi.com

Principal Investigator

Clinical Sciences & Operations

STUDY_DIRECTOR

Sanofi

Study Locations (Sites)

Children's Hospital Los Angeles- Site Number : 8400005

Los Angeles, California, 90027

United States

Collaborators and Investigators

Sponsor: Sanofi

Clinical Sciences & Operations, STUDY_DIRECTOR, Sanofi

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2024-03-01

Study Completion Date2029-11-30

Study Record Updates

Study Start Date2024-03-01

Study Completion Date2029-11-30

Terms related to this study

Additional Relevant MeSH Terms

Hemophilia A