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

Completed

• Conditions: Hemophilia A

• Registration Number: NCT04560933
• Lead Sponsor: BioMarin Pharmaceutical

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 Timeline

• Study Start: 2020-08-25
• Study First Submitted: 2020-09-15
• Study First Submitted QC: 2020-09-22
• Study First Posted: 2020-09-23
• Primary Completion: 2023-09-20
• Study Completion: 2023-10-20
• Status Verified: 2024-04
• Last Update Submitted: 2024-04-22
• Last Update Posted: 2024-04-24

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 186

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

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A	Baseline	To assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A at Baseline
Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A	Change from baseline through Week 12

Secondary Outcome Measures

Name	Time	Method
To describe and characterize AAV vector titer values in subjects with Hemophilia A	Change from baseline through Week 24	To describe and characterize AAV vector titer values in subjects with Hemophilia A

Trial Locations

Locations (1)

Covance Inc; 🇺🇸 Madison, Wisconsin, United States