Phase 3 Haemophilia Trial, Recruiting

Written by an AI model from ClinicalTrials.gov eligibility criteria and checked on a sample basis. Please confirm against the original criteria.

Who might be able to join this trial:

• Children who are at least 1 year old and under 12 years old at the time of joining the study
• Children who have been diagnosed with severe hemophilia A or B, confirmed by a blood test or medical records
• Children with hemophilia who have developed "inhibitors" (antibodies that block standard clotting treatments) and have been using bypass agents to control or prevent bleeding for at least the 3 months before screening, with blood test results confirming the inhibitor, OR children without inhibitors who have been using clotting factor concentrates for at least 3 months before screening and have not needed bypass agents during that time
• Children who have suitable veins to allow the blood draws required by the study, as assessed by the study doctor
• A parent or legal guardian must be willing and able to provide signed consent on the child's behalf, and the child must provide their own agreement to participate as required by local rules

Who may not be able to join:

• Children who have another bleeding disorder in addition to hemophilia A or B
• Children with significant liver disease
• Children with a history of antiphospholipid antibody syndrome (a condition affecting blood clotting)
• Children who have had blood clots in arteries or veins unrelated to an indwelling venous access device
• Children with any other medical condition that the study doctor believes would make participation unsafe or interfere with the study
• Children with a history of allergies to multiple medications, or a known allergic reaction to a type of molecule called an oligonucleotide or GalNAc (confirm with trial site)
• Children who have an indwelling catheter and have had serious venous access complications such as infections or blood clots requiring hospitalisation or blood-thinning treatment in the past 12 months
• Children expected to need surgery at any point during the study, or who have had surgery within 14 days before screening
• Children who are currently receiving extra bypass agent infusions to manage bleeding after a recent surgery
• Children who have had a bad reaction to injections under the skin (subcutaneous injections)
• Children currently undergoing immune tolerance induction (ITI) therapy, which is a treatment to reduce inhibitors
• Children who have used emicizumab (Hemlibra®) or similar non-factor bleed management treatments within 6 months before screening
• Children who have previously received gene therapy
• Children currently participating in, or planning to participate in, another clinical trial involving a different investigational treatment or device during this study
• Children whose antithrombin activity level is below 60% at screening (a blood measurement related to clotting, confirmed by a central laboratory)
• Children with an existing condition that increases their risk of blood clots (thrombophilic disorder)
• Children with an active Hepatitis C virus infection
• Children with an active Hepatitis A or Hepatitis E infection
• Children with an active or ongoing Hepatitis B infection
• Children with a platelet count at or below 100,000 per microlitre of blood
• Children with an active infection at the time of screening
• Children who are HIV positive with a CD4 cell count below 400 cells per microlitre
• Children with reduced kidney function below a certain level, as measured by a specific calculation called the Schwartz formula

Important: Always verify eligibility with the trial site directly before applying.