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Clinical Trial 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

Clinical Trial 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. ;

Study Design

Related Conditions & MeSH terms

NCT number NCT04560933
Study type Observational
Source BioMarin Pharmaceutical
Status Completed
Start date August 25, 2020
Completion date October 20, 2023

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