Brief Title
A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
Official Title
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
Brief 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.
Study Type
Observational
Primary Outcome
Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A
Secondary Outcome
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Condition
Hemophilia A
Intervention
Blood sample collection
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Procedure
Estimated Enrollment
1000
Start Date
August 25, 2020
Completion Date
April 30, 2023
Primary Completion Date
March 30, 2023
Eligibility Criteria
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
Gender
All
Ages
18 Years - N/A
Contacts
Stephen Flach, MD, +447823336138, [email protected]
Location Countries
United States
Location Countries
United States
Administrative Informations
NCT ID
NCT04560933
Organization ID
270-701
Responsible Party
Sponsor
Study Sponsor
BioMarin Pharmaceutical
Study Sponsor
Stephen Flach, MD, Principal Investigator, Covance
Verification Date
September 2022