What Screenings May Be Required?

What screenings may be required for an investigational gene therapy clinical trial for hemophilia?

Multiple investigational gene therapy clinical trials for hemophilia are currently underway, and each one is different. Before beginning a clinical trial, potential participants are screened to determine who is eligible (inclusion criteria) or ineligible (exclusion criteria). Because clinical trials differ, they typically have different screenings. That means if you are not eligible for one clinical trial, you may still be eligible for another.

Common screenings for a hemophilia gene therapy clinical trial may include:

Age (eg, 18 years or older)

Age (eg, 18 years or older)

Severity of hemophilia

Severity of hemophilia

Factor inhibitor status

Factor inhibitor status

Overall health history, including health of the liver

Overall health history
(ie, health of the liver)

Pre-existing neutralizing antibodies (NAbs)

Pre-existing neutralizing
antibodies (NAbs)

These are just some of the screenings a gene therapy clinical trial for hemophilia may review. Every clinical trial is different, so you should talk to your doctor and healthcare team if you are considering participating in one to make sure you understand it.