Recruiting

Testing a new bleeding treatment for young boys with hemophilia

Plain-English translation of NCT07285460 on ClinicalTrials.gov ↗ · Source last updated August 2026 · Translation generated August 2026 · How we translate trials

What is this trial?

This trial is testing a medication called to help young boys (ages 1 to 12) with hemophilia A or B—a bleeding disorder where the body struggles to form blood clots. Some children in this study may have developed antibodies that block standard clotting treatments, making bleeding even harder to control. The medication works differently than older treatments and is being studied to see if it can reduce bleeding and be safely used in this young age group.

Children with hemophilia, especially those who have developed antibodies to standard clotting medicines, have few good treatment options and face serious risks from uncontrolled bleeding. This trial aims to find out whether this new medication can help these children bleed less and live safer, more active lives.

Do you qualify?

You likely qualify if…

you are a boy between 1 and 12 years old
you have been diagnosed with severe hemophilia A or B (a bleeding disorder confirmed by blood tests)
you have been using clotting medicines regularly for at least 3 months before joining
you either have inhibitory antibodies to clotting factors (and have been using bypass agents) or you don't have these antibodies (and have been using standard clotting medicines)
you have good access to veins for the blood draws the study requires
your parent or legal guardian can give informed consent and you can give your assent to participate

You likely don't qualify if…

you have other bleeding disorders besides hemophilia A or B
you have significant liver disease or a history of blood clots in your veins or arteries
you have had a severe allergic reaction to similar types of medicines in the past
you currently have a central or peripheral catheter (permanent tube in your vein) or a history of serious problems from venous access
you have antiphospholipid antibody syndrome or any condition that the study doctor believes would make participation unsafe or difficult

What participation looks like

If you're new to this medication, you'll first receive standard care for 24 weeks to establish a baseline, then begin receiving the medication on a regular schedule for up to 160 weeks total (about 3 years). If you're already taking this medication from a related study, you'll continue your current dose and may be adjusted as needed. Throughout the study, you'll have regular clinic visits for blood draws and check-ins to monitor how well the medication is working and ensure it's safe.

Testing a new bleeding treatment for young boys with hemophilia

What is this trial?

Do you qualify?

What participation looks like

Trial locations(30 sites)