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Clinical Trial Details — Status: Completed

Administrative data

NCT number NCT04560933
Other study ID # 270-701
Secondary ID
Status Completed
Phase
First received
Last updated
Start date August 25, 2020
Est. completion date October 20, 2023

Study information

Verified date April 2024
Source BioMarin Pharmaceutical
Contact n/a
Is FDA regulated No
Health authority
Study type Observational

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


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.


Recruitment information / eligibility

Status Completed
Enrollment 186
Est. completion date October 20, 2023
Est. primary completion date September 20, 2023
Accepts healthy volunteers
Gender All
Age group 18 Years and older
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

Study Design


Related Conditions & MeSH terms


Intervention

Procedure:
Blood sample collection
Blood sample collection

Locations

Country Name City State
United States Covance Inc Madison Wisconsin

Sponsors (1)

Lead Sponsor Collaborator
BioMarin Pharmaceutical

Country where clinical trial is conducted

United States, 

Outcome

Type Measure Description Time frame Safety issue
Primary Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A To assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A at Baseline Baseline
Primary Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A Change from baseline through Week 12
Secondary To describe and characterize AAV vector titer values in subjects with Hemophilia A To describe and characterize AAV vector titer values in subjects with Hemophilia A Change from baseline through Week 12
Secondary To describe and characterize AAV vector titer values in subjects with Hemophilia A To describe and characterize AAV vector titer values in subjects with Hemophilia A Change from baseline through Week 24
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