Skip to main content
OpenTrials
Completed

NCT Number: NCT04560933

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

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

Completed

Looking for future studies?

Notify Me

Key information

Conditions

Hemophilia A Blood Coagulation Disorders Blood Coagulation Disorders, Inherited Coagulation Protein Disorders Congenital, Hereditary, and Neonatal Diseases and Abnormalities Genetic Diseases, Inborn Hematologic Diseases Hemic and Lymphatic Diseases Hemorrhagic Disorders

Age range

18 year and older

Sex eligibility

All sexes

Study type

Observational

Primary location

Covance Inc

Madison, Wisconsin, 53704, United States

About this study

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.

Who can participate

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

blood sample collection

Procedure

Blood sample collection

Primary outcomes

  1. 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

  2. Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A

    Time frame: Change from baseline through Week 12

Secondary outcomes

  1. 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

  2. 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

Sponsors and collaborators

Lead sponsor

BioMarin Pharmaceutical

Industry

Registry information

Official study 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

Acronym: SAAVY

Important dates

Study start
2020
Primary completion
2023
Study completion
2023
First posted
Sep 23, 2020
Registry last updated
Apr 24, 2024

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

Related clinical trials

Published trials that share one or more normalized conditions with this study.