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Tests treatment safety and results for Hemophilia
Official title A Study to Investigate the Efficacy and Safety of Fitusiran Prophylaxis in Male Participants Aged 1 to Less Than 12 Years With Hemophilia A or B
ClinicalTrials.gov ID: NCT07285460
What this study is testing
What is Fitusiran?
Fitusiran is an investigational medicine, given as an injection under the skin, being studied as a potential treatment for hemophilia.
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 a parallel, Phase 3, two-arm, open-label study to evaluate the efficacy and safety of treatment with fitusiran prophylaxis administered to male pediatric participants (aged 1 to \<12 years) who have severe hemophilia A or B, with or without inhibitory antibodies to FVIII or FIX. Number of participants: Approximately 85 participants will be enrolled into the study: Approximately 60 fitusiran-naïve participants with severe hemophilia A or B, with or without inhibitors (fitusiran-naïve arm), and Approximately 25 participants with severe hemophilia A or B with inhibitors rolling over from the EFC15467\ dose confirmation study (roll-over arm).
- Phase 3: a large, late-stage study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 1 to 11, men only
You may be able to join if
- Participants not previously exposed to fitusiran are eligible to be included in the study only if all of the following criteria apply:
- Participant must be 1 to \<12 years of age at the time of enrollment.
- Participants must have severe hemophilia A or B (FVIII \<1% or FIX ≤2%) as evidenced by a central laboratory measurement at screening or documented...
- Participants must meet inhibitor or non-inhibitor status as defined below: Inhibitor: Requiring use of BPA for prophylaxis or BPA as on-demand...
- Inhibitor titer of ≥0.6 BU/mL at screening, OR
You likely can't join if
- Participants not previously exposed to fitusiran are excluded from the study if any of the following criteria apply:
- Known co-existing bleeding disorders other than hemophilia A or B.
- Presence of clinically significant liver disease.
- History of antiphospholipid antibody syndrome.
- History of arterial or venous thromboembolism, unrelated to an indwelling venous access
- Any condition (eg, medical concern), which in the opinion of the Investigator, would make the participant unsuitable for dosing or which could...
See the full eligibility criteria
- Participants not previously exposed to fitusiran are eligible to be included in the study only if all of the following criteria apply:
- Participant must be 1 to \<12 years of age at the time of enrollment.
- Participants must have severe hemophilia A or B (FVIII \<1% or FIX ≤2%) as evidenced by a central laboratory measurement at screening or documented medical record evidence.
- Participants must meet inhibitor or non-inhibitor status as defined below: Inhibitor: Requiring use of BPA for prophylaxis or BPA as on-demand therapy for any bleeding episodes for at least the last 3 months prior to...
- 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 1 inhibitor titer ≥0.6 BU/mL and a history of anamnestic response, or severe allergic reaction (eg, anaphylaxis) or nephrotic syndrome...
- Nijmegen-modified Bethesda assay inhibitor titer of \<0.6 BU/mL at screening, AND
- No use of BPA to treat bleeding episodes for at least the last 3 months prior to screening
- Participants must have adequate peripheral venous access, as determined by the Investigator, to allow the blood draws required by the study protocol.
- Male: There are no contraceptive requirements for this study except where required by local regulations.
- Capable of giving signed informed consent/assent. A signed written informed consent must be obtained from parent(s)/legal guardian (hereafter referred to as the "parent"), as well as a written or oral assent obtained...
- Participants not previously exposed to fitusiran are excluded from the study if any of the following criteria apply:
- Known co-existing bleeding disorders other than hemophilia A or B.
- Presence of clinically significant liver disease.
- History of antiphospholipid antibody syndrome.
- History of arterial or venous thromboembolism, unrelated to an indwelling venous access
- Any condition (eg, medical concern), which in the opinion of the Investigator, would make the participant unsuitable for dosing or which could interfere with the study compliance, the participant's safety and/or the...
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
- people with a central or peripheral indwelling catheter, with a history of venous access complications (such as infections, thrombosis) leading to hospitalization and/or systemic anticoagulation therapy in the last 12...
- At screening, anticipated need of surgery during the study or planned surgery scheduled to occur during the study.
- Completion of a surgical procedure within 14 days prior to screening, or currently receiving additional BPA infusion for postoperative hemostasis.
- History of intolerance to SC injection(s).
- Current participation in ITI therapy.
- The use of emicizumab (Hemlibra®) or any non-factor bleed management treatment within 6 months prior to screening
- Prior gene therapy
- Current or future participation in another clinical study, scheduled to occur during this study, involving an investigational product other than fitusiran or an investigational device.
- AT activity \<60% at screening, as determined by central laboratory analysis.
- Co-existing thrombophilic disorder.
- Presence of an active Hepatitis C virus infection
- Presence of acute hepatitis A or Hepatitis E virus infection.
- Presence of acute or chronic hepatitis B virus infection.
- Platelet count ≤100 000/μL.
- Presence of acute infection at screening.
- Human immunodeficiency virus (HIV) positive with a CD4 count of \<400 cells/μL.
- Estimated glomerular filtration rate ≤45 mL/min/1.73 m2 (using the Schwartz formula). The above information is not intended to contain all considerations relevant to a patient's potential participation in a clinical...
The study team makes the final eligibility decision.
Where it's taking place
- Los Angeles, California, United States
- Orange, California, United States
- Las Vegas, Nevada, United States
- Hackensack, New Jersey, United States
- Dallas, Texas, United States
- Brussels, Belgium
- Ribeirão Preto, São Paulo, Brazil
- São Paulo, Brazil
- Hamilton, Ontario, Canada
- Montreal, Quebec, Canada
- Beijing, China
- Bengaluru, India
- Mumbai, India
- Pune, India
- Vellore, India
- Florence, Firenze, Italy
- Milan, Milano, Italy
- Rozzano, Milano, Italy
- Wroclaw, Lower Silesian Voivodeship, Poland
- Iași, Romania
+ 9 more site(s).
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, 1 year to 11 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Los Angeles, California, United States; Orange, California, United States; Las Vegas, Nevada, United States; Hackensack, New Jersey, United States; Dallas, Texas, United States; Brussels, Belgium and 23 more location(s). 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.