Trial studying safety and effects 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?
An investigational subcutaneous injectable medicine being studied as a potential treatment for severe hemophilia A or B in children. It is designed to lower antithrombin levels in the blood, which can increase clotting activity.
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 study is testing whether fitusiran works well and is safe. It also looks at any side effects the medicine may cause.
- Who it's for
- You may be able to join if you are a boy aged 1 to 11 with severe hemophilia A or B.
- What's involved
- You would get fitusiran shots to help prevent bleeding. You would also have regular check-ups and blood tests.
- Time commitment
- The study lasts up to 160 weeks for new patients or 60 weeks for others.
- 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
- 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...
- 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
- Nijmegen-modified Bethesda assay inhibitor titer of \<0.6 BU/mL at screening, AND
You likely can't join if
- 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...
- History of multiple drug allergies or history of allergic reaction to an oligonucleotide or GalNAc.
See the full eligibility criteria
- 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.
- 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
- 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...
- 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 is enrolling
- Los Angeles, California, United States
- Orange, California, United States
- Torrance, California, United States
- Ann Arbor, Michigan, United States
- Las Vegas, Nevada, United States
- Hackensack, New Jersey, United States
- Dallas, Texas, United States
- Brussels, Belgium
- Campinas, São Paulo, Brazil
- Ribeirão Preto, São Paulo, Brazil
- São Paulo, Brazil
- Hamilton, Ontario, Canada
- Toronto, Ontario, Canada
- Montreal, Quebec, Canada
- Beijing, China
- Wuhan, China
- Bengaluru, India
- Mumbai, India
- Pune, India
- Vellore, India
+ 15 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, but 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?
This study is enrolling now at Los Angeles, California, United States; Orange, California, United States; Torrance, California, United States; Ann Arbor, Michigan, United States; Las Vegas, Nevada, United States; Hackensack, New Jersey, United States and 29 more locations. Enter your location above to see the nearest enrolling site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool that is independent of the study sponsor. Details come from ClinicalTrials.gov. The study team decides eligibility, and your own doctor gives medical advice.