NCT04560933

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

A Prospective, Observational Study Evaluating Seroprevalence and Rate of Seroconversion of Antibodies Against Adeno-associated Virus (AAV) Serotypes and Exploratory Vectors in Subjects With Hemophilia A in the United States

• Status: Completed
• Phase: —
• Study type: Observational
• Enrollment: 186 (actual)
• Sponsor: BioMarin Pharmaceutical · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: —

Summary

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

Detailed 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.

Conditions

• Hemophilia A

Interventions

Type	Name	Description
PROCEDURE	Blood sample collection	Blood sample collection

Timeline

• Start date: 2020-08-25
• Primary completion: 2023-09-20
• Completion: 2023-10-20
• First posted: 2020-09-23
• Last updated: 2024-04-24

Locations

1 site across 1 country: United States