Frequently Asked Questions

All children who qualify for the study will receive the study drug for 12 months.

“Study drug” means that the medicine is currently not approved by country-specific regulatory health authorities to be used to treat a disease but is allowed to be used for clinical research purposes.

Like with all medicines, there is a chance for side effects, which are unwanted and unintended effects, from the study drug. Your health will be closely monitored during the entire study. It is very important to tell the study doctor if you notice any changes in your child’s health or if anything is bothering him.

No. You will use your child’s usual prescribed hemophilia medication to treat bleeds when they occur. The study doctor will let you know how much medication to use and when.

Your child will be in the study for approximately 14 months.

Your child will visit the study site a total of about 10 times for tests (this includes the visit to check your child’s eligibility). Some of the visits may take place at your home if you prefer and if local regulations allow it. Your child will also have about 6 phone calls with the study team to check on his health (5 phone calls while taking the study drug and 1 phone call after completing treatment).

Your child may or may not directly benefit from being in the clinical study. Your child will have continuous monitoring from doctors with expertise in hemophilia, and any information collected during the study may help doctors learn more about treating other children with hemophilia.

Having your child take part in the study will not cost you anything. Your child will receive the study drug and study tests at no cost to you.

After your child completes the study, he may have the option to continue taking the study drug for an extended period of time as part of a separate study. You can discuss this with your child’s study doctor if you are interested.

Being in the study is completely voluntary. If your child joins and then changes his mind, he can leave the study at any time.