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

Plain-language summary

This observational study set out to quantify how common antibodies to the adeno-associated virus (AAV) types AAV5, AAV6, and AAV8 are (seroprevalence) in people with hemophilia A, and how often such antibodies develop (seroconversion) over varying follow-up intervals. The only procedure is blood sample collection; no treatment is given. It enrolled people aged 18 or over with a diagnosis of hemophilia A who were not taking part in another interventional study and had not previously received AAV vector gene therapy.

The summary above is a plain-language rendering of the official record. The original English title is shown for reference.

Trial details

Phase

N/A

Status

Completed

Sponsor

BioMarin Pharmaceutical

Intervention

Blood sample collection

Start date

8/25/2020

Estimated completion

10/20/2023

Countries

United States

Related genes

Locations

• Madison, United States

Eligibility criteria

Criteria are reproduced from the ClinicalTrials.gov record in the original English. Only the trial team can determine eligibility.

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