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

Sponsor

BioMarin Pharmaceutical (Industry)

Overall Status

Not yet recruiting

CT.gov ID

NCT05580692

Collaborator

(none)

400

Enrollment

Arm

Anticipated Duration (Months)

Study Details

Study Description

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

Condition or Disease	Intervention/Treatment	Phase
Hemophilia A	Procedure: Biospecimen Collection	N/A

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 Design

Study Type:

Interventional

Anticipated Enrollment :

400 participants

Allocation:

N/A

Intervention Model:

Single Group Assignment

Masking:

None (Open Label)

Primary Purpose:

Other

Official Title:

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

Anticipated Study Start Date :

Oct 1, 2022

Anticipated Primary Completion Date :

Oct 1, 2025

Anticipated Study Completion Date :

Nov 1, 2025

Arms and Interventions

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

Arm

Intervention/Treatment

Experimental: Patient Cohort

All Patients are within the same arm

Procedure: Biospecimen Collection

Biospecimen Sample Collection

Outcome Measures

Primary Outcome Measures

To qunatify Seroprevalence of antibodies to selected AAV serotypes [52 weeks]
Number of patients with detectable antibodies to selected AAV serotypes

Secondary Outcome Measures

To describe and characterize selected AAV serotypes antibody responses in adult subjects with Hemophilia A [104 weeks]
Antibody titer levels measured in target population to selected AAV serotypes

Eligibility Criteria

Criteria

Ages Eligible for Study:

18 Years and Older

Sexes Eligible for Study:

All

Accepts Healthy Volunteers:

Inclusion Criteria:

Adult Subjects with Hemophilia A

Exclusion Criteria:

Subject previously treated with AAV vector gene therapy

Contacts and Locations

Locations

No locations specified.

Sponsors and Collaborators

BioMarin Pharmaceutical

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

BioMarin Pharmaceutical

ClinicalTrials.gov Identifier:

NCT05580692

Other Study ID Numbers:

270-702

First Posted:

Oct 14, 2022

Last Update Posted:

Oct 14, 2022

Last Verified:

Jul 1, 2022

Individual Participant Data (IPD) Sharing Statement:

Plan to Share IPD:

Studies a U.S. FDA-regulated Drug Product:

Studies a U.S. FDA-regulated Device Product:

Additional relevant MeSH terms:

Hemophilia A

Study Results

No Results Posted as of Oct 14, 2022