A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)

August 15, 2024 updated by: BioMarin Pharmaceutical

A Multinational, Multi-center, Prospective Cohort Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV) Serotypes in Subjects With Hemophilia A

A prospective cohort study utilizing biospecimen sample collection from adult Hemophilia A subjects to evaluate and characterize seroprevalence and the seroconversion of antibodies against AAV serotypes

Study Overview

Status

Terminated

Conditions

Intervention / Treatment

Detailed Description

This is a multinational, multi-center, prospective cohort study utilizing biospecimen sample collection from adult Hemophilia A subjects to evaluate and characterize seroprevalence and the seroconversion of antibodies against AAV serotypes

Study Type

Interventional

Enrollment (Actual)

321

Phase

  • Not Applicable

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

  • Argentina
      • Corrientes, Argentina, W3410 FND
        • Hospital Ramon Jose Vidal
      • Mendoza, Argentina
        • Medico Hematologo
      • Rosario, Argentina, S2000 CFK
        • Instituto de Hematologia y Medicina Clinica Dr. RUben Davoli
    • Mendoza
      • Godoy Cruz, Mendoza, Argentina, M5504FKD
        • Instituto Hematologia Arbesu
  • Australia
      • Camperdown, Australia, 2050
        • Royal Prince Alfred Hospital
      • Murdoch, Australia, 6150
        • Haemophilia and Haemostasis Centre Level 1 Cancer Centre South Metropolitan Health Service Fiona Stanley Hospital
  • Austria
      • Wien, Austria, 1090
        • Medical University of Vienna
  • Saudi Arabia
      • Riyadh, Saudi Arabia, 11211
        • King Faisal Specialist Hospital & Research Center
      • Riyadh, Saudi Arabia, 12372
        • King Saud University Medical City King Saud University
  • Sweden
      • Malmö, Sweden, 205 02
        • Skane University Hospital
      • Solna, Sweden, SE-171 76
        • ME Centrum för kliniska cancerstudier - House H3
  • Turkey
      • Adana, Turkey, 01130
        • Acibadem Adana Hospital
      • Ankara, Turkey, 06100
        • Hacettepe University
      • Gaziantep, Turkey, 27310
        • Gaziantep University Medical Faculty Sahinbey Educational Research Hospital
      • Istanbul, Turkey, 34098
        • Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty
      • Samsun, Turkey, 55270
        • Ondokuz Mayis Univ. Med. Fac.
      • İzmir, Turkey, 35040
        • Ege University Medical Faculty
  • United Arab Emirates
      • Abu Dhabi, United Arab Emirates, 11393
        • Sheikh Shakhbout Medical City

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

No

Description

Inclusion Criteria:

  • Adult Subjects with Hemophilia A

Exclusion Criteria:

  • Subject 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

  • Primary Purpose: Other
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Patient Cohort
All Patients are within the same arm
Procedure: Biospecimen Collection
Biospecimen Sample Collection

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To qunatify Seroprevalence of antibodies to selected AAV serotypes
Time Frame: 52 weeks
Number of patients with detectable antibodies to selected AAV serotypes
52 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
To describe and characterize selected AAV serotypes antibody responses in adult subjects with Hemophilia A
Time Frame: 52 weeks
Antibody titer levels measured in target population to selected AAV serotypes
52 weeks

Collaborators and Investigators

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

Sponsor

BioMarin Pharmaceutical

Investigators

  • Study Director: Medinfo Medinfo, BioMarin Pharmaceutical

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)

March 14, 2023

Primary Completion (Actual)

July 31, 2024

Study Completion (Actual)

July 31, 2024

Study Registration Dates

First Submitted

September 16, 2022

First Submitted That Met QC Criteria

October 11, 2022

First Posted (Actual)

October 14, 2022

Study Record Updates

Last Update Posted (Actual)

August 19, 2024

Last Update Submitted That Met QC Criteria

August 15, 2024

Last Verified

August 1, 2024

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • 270-702

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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