A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A (SAAVY)

BioMarin Pharmaceutical

Status: Completed

Conditions: Hemophilia A

Treatments: Procedure: Blood sample collection

Study type: Observational

Funder types: Industry

Identifiers

NCT04560933

270-701

Details and patient eligibility

About

To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A

Full description

This is a single-center, decentralized, patient-centered, prospective, observational study utilizing biospecimen samples collected from hemophilia A subjects across the United States to evaluate and characterize seroprevalence and the rate of seroconversion of antibodies against AAV serotypes and exploratory vectors, and to investigate the associated factors that may influence the vector titers.

Relevant medical findings will also be collected from the subject, as well as symptoms related to hemophilia A. The collection of medical history may include major illnesses, diagnoses, and surgeries.

Enrollment: 186 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Subjects diagnosed with Hemophilia A
• Subjects aged 18years or over at time of entry

Exclusion criteria

• Currently participating in an interventional study of any investigational product, device or procedure.
• Subjects who have been previously treated with AAV vector gene therapy