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New treatment option for Hemophilia A
Official title A Study to Test a Medicine (Fitusiran) for Preventing Bleeds in People With Severe Hemophilia Who Previously Received Preventive Treatment With Emicizumab
ClinicalTrials.gov ID: NCT06145373
What this study is testing
What is Fitusiran (SAR439774)?
Fitusiran (SAR439774) is an investigational medicine, given as a monthly injection under the skin, being studied as a potential treatment for hemophilia a.
Also referred to as SAR439774, Qfitlia®.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- 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.
- Phase 4: studies an already-approved treatment
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 12 and older, men only
You may be able to join if
- 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...
- 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.
You likely can't join if
- Participants are excluded from the study if any of the following criteria apply:
- 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...
- Presence of clinically significant liver disease
- Current or prior participation in a fitusiran trial
See the full eligibility 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.
- Participants are excluded from the study if any of the following criteria apply:
- 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...
- 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 The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical trial.
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Taipei, Podlaskie, Taiwan
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling male, 12 years and older. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Taipei, Podlaskie, Taiwan. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.