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

April 22, 2024 updated by: BioMarin Pharmaceutical

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

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

Study Overview

Status

Completed

Conditions

Intervention / Treatment

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

Enrollment (Actual)

186

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Wisconsin
      • Madison, Wisconsin, United States, 53704
        • Covance Inc

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

N/A

Sampling Method

Non-Probability Sample

Study Population

Subjects diagnosed with Hemophilia A

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A
Time Frame: Baseline
To assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A at Baseline
Baseline
Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A
Time Frame: Change from baseline through Week 12
Change from baseline through Week 12

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Time Frame: Change from baseline through Week 12
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Change from baseline through Week 12
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Time Frame: Change from baseline through Week 24
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Change from baseline through Week 24

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

BioMarin Pharmaceutical

Investigators

  • Principal Investigator: Stephen Flach, MD, Covance

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

August 25, 2020

Primary Completion (Actual)

September 20, 2023

Study Completion (Actual)

October 20, 2023

Study Registration Dates

First Submitted

September 15, 2020

First Submitted That Met QC Criteria

September 22, 2020

First Posted (Actual)

September 23, 2020

Study Record Updates

Last Update Posted (Actual)

April 24, 2024

Last Update Submitted That Met QC Criteria

April 22, 2024

Last Verified

April 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 270-701

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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