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

Not Applicable

Terminated

• Conditions: Hemophilia A
• Interventions: Procedure: Biospecimen Collection

• Registration Number: NCT05580692
• Lead Sponsor: BioMarin Pharmaceutical

Brief Summary

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

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 Timeline

• Study First Submitted: 2022-09-16
• Study First Submitted QC: 2022-10-11
• Study First Posted: 2022-10-14
• Study Start: 2023-03-14
• Primary Completion: 2024-07-31
• Study Completion: 2024-07-31
• Status Verified: 2024-08
• Last Update Submitted: 2024-08-15
• Last Update Posted: 2024-08-19

Recruitment & Eligibility

• Status: TERMINATED
• Sex: All
• Target Recruitment: 321

Inclusion Criteria

• Adult Subjects with Hemophilia A

Exclusion Criteria

• Subject previously treated with AAV vector gene therapy

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Patient Cohort	Biospecimen Collection	All Patients are within the same arm

Primary Outcome Measures

Name	Time	Method
To qunatify Seroprevalence of antibodies to selected AAV serotypes	52 weeks	Number of patients with detectable antibodies to selected AAV serotypes

Secondary Outcome Measures

Name	Time	Method
To describe and characterize selected AAV serotypes antibody responses in adult subjects with Hemophilia A	52 weeks	Antibody titer levels measured in target population to selected AAV serotypes

Trial Locations

Locations (18)

King Faisal Specialist Hospital & Research Center; 🇸🇦 Riyadh, Saudi Arabia

Instituto de Hematologia y Medicina Clinica Dr. RUben Davoli; 🇦🇷 Rosario, Argentina

Instituto Hematologia Arbesu; 🇦🇷 Godoy Cruz, Mendoza, Argentina

Hospital Ramon Jose Vidal; 🇦🇷 Corrientes, Argentina

Medico Hematologo; 🇦🇷 Mendoza, Argentina

Haemophilia and Haemostasis Centre Level 1 Cancer Centre South Metropolitan Health Service Fiona Stanley Hospital; 🇦🇺 Murdoch, Australia

Royal Prince Alfred Hospital; 🇦🇺 Camperdown, Australia

Medical University of Vienna; 🇦🇹 Wien, Austria

King Saud University Medical City King Saud University; 🇸🇦 Riyadh, Saudi Arabia

Skane University Hospital; 🇸🇪 Malmö, Sweden

Acibadem Adana Hospital; 🇹🇷 Adana, Turkey

ME Centrum för kliniska cancerstudier - House H3; 🇸🇪 Solna, Sweden

Hacettepe University; 🇹🇷 Ankara, Turkey

Sheikh Shakhbout Medical City; 🇦🇪 Abu Dhabi, United Arab Emirates

Gaziantep University Medical Faculty Sahinbey Educational Research Hospital; 🇹🇷 Gaziantep, Turkey

Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty; 🇹🇷 Istanbul, Turkey

Ondokuz Mayis Univ. Med. Fac.; 🇹🇷 Samsun, Turkey

Ege University Medical Faculty; 🇹🇷 İzmir, Turkey