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

NCT05580692 | Terminated

• 1 other identifier: 270-702
• Study Type: interventional
• Target: 321
• Locations: 6 countries
• Sites: 16

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

Conditions

Hemophilia A

Outcome Measures

Primary Outcomes (1)

• To qunatify Seroprevalence of antibodies to selected AAV serotypes

  Number of patients with detectable antibodies to selected AAV serotypes

  52 weeks

Secondary Outcomes (1)

• To describe and characterize selected AAV serotypes antibody responses in adult subjects with Hemophilia A

  52 weeks

Study Arms (1)

Patient Cohort

EXPERIMENTAL

All Patients are within the same arm

Procedure: Biospecimen Collection

Interventions

Biospecimen Collection PROCEDURE

Biospecimen Sample Collection

Patient Cohort

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Adult Subjects with Hemophilia A

You may not qualify if:

• Subject previously treated with AAV vector gene therapy

Sponsors & Collaborators

• BioMarin Pharmaceutical: lead

Study Sites (18)

Instituto Hematologia Arbesu

Godoy Cruz, Mendoza Province, M5504FKD, Argentina

Location

Hospital Ramon Jose Vidal

Corrientes, W3410 FND, Argentina

Location

Medico Hematologo

Mendoza, Argentina

Location

Instituto de Hematologia y Medicina Clinica Dr. RUben Davoli

Rosario, S2000 CFK, Argentina

Location

Royal Prince Alfred Hospital

Camperdown, 2050, Australia

Location

Haemophilia and Haemostasis Centre Level 1 Cancer Centre South Metropolitan Health Service Fiona Stanley Hospital

Murdoch, 6150, Australia

Location

Medical University of Vienna

Vienna, 1090, Austria

Location

King Faisal Specialist Hospital & Research Center

Riyadh, 11211, Saudi Arabia

Location

King Saud University Medical City King Saud University

Riyadh, 12372, Saudi Arabia

Location

Skane University Hospital

Malmo, 205 02, Sweden

Location

ME Centrum för kliniska cancerstudier - House H3

Solna, SE-171 76, Sweden

Location

Acibadem Adana Hospital

Adana, 01130, Turkey (Türkiye)

Location

Hacettepe University

Ankara, 06100, Turkey (Türkiye)

Location

Gaziantep University Medical Faculty Sahinbey Educational Research Hospital

Gaziantep, 27310, Turkey (Türkiye)

Location

Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty

Istanbul, 34098, Turkey (Türkiye)

Location

Ege University Medical Faculty

Izmir, 35040, Turkey (Türkiye)

Location

Ondokuz Mayis Univ. Med. Fac.

Samsun, 55270, Turkey (Türkiye)

Location

Sheikh Shakhbout Medical City

Abu Dhabi, 11393, United Arab Emirates

Location

MeSH Terms

Conditions

Hemophilia A

Condition Hierarchy (Ancestors)

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

Study Officials

• Medinfo Medinfo

  BioMarin Pharmaceutical

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: not applicable
• Allocation: NA
• Masking: NONE
• Purpose: OTHER
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 16, 2022
• First Posted: October 14, 2022
• Study Start: March 14, 2023
• Primary Completion: July 31, 2024
• Study Completion: July 31, 2024
• Last Updated: August 19, 2024

Record last verified: 2024-08

Data Sharing

• IPD Sharing: Will not share

Locations

AR (4); SE (2); TU (6); SA (2); AE (1); AU (1)