Back to resultsA Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
Primary Purpose
Hemophilia A
Status
Recruiting
Phase
Not Applicable
Locations
Argentina
Study Type
Interventional
Intervention
Biospecimen Collection
Sponsored by
About this trial
This is an interventional other trial for Hemophilia A
Eligibility Criteria
Inclusion Criteria:
- Adult Subjects with Hemophilia A
Exclusion Criteria:
- Subject previously treated with AAV vector gene therapy
Sites / Locations
- Medico HematologoRecruiting
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
Patient Cohort
Arm Description
All Patients are within the same arm
Outcomes
Primary Outcome Measures
To qunatify Seroprevalence of antibodies to selected AAV serotypes
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
Antibody titer levels measured in target population to selected AAV serotypes
Full Information
NCT ID
NCT05580692
First Posted
September 16, 2022
Last Updated
April 24, 2023
Sponsor
BioMarin Pharmaceutical
1. Study Identification
Unique Protocol Identification Number
NCT05580692
Brief Title
A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
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
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Recruiting
Study Start Date
March 14, 2023 (Actual)
Primary Completion Date
October 2025 (Anticipated)
Study Completion Date
November 2025 (Anticipated)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
BioMarin Pharmaceutical
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
No
5. 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
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Hemophilia A
7. Study Design
Primary Purpose
Other
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
400 (Anticipated)
8. Arms, Groups, and Interventions
Arm Title
Patient Cohort
Arm Type
Experimental
Arm Description
All Patients are within the same arm
Intervention Type
Procedure
Intervention Name(s)
Biospecimen Collection
Intervention Description
Biospecimen Sample Collection
Primary Outcome Measure Information:
Title
To qunatify Seroprevalence of antibodies to selected AAV serotypes
Description
Number of patients with detectable antibodies to selected AAV serotypes
Time Frame
52 weeks
Secondary Outcome Measure Information:
Title
To describe and characterize selected AAV serotypes antibody responses in adult subjects with Hemophilia A
Description
Antibody titer levels measured in target population to selected AAV serotypes
Time Frame
104 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
Adult Subjects with Hemophilia A
Exclusion Criteria:
Subject previously treated with AAV vector gene therapy
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
MedInfo
Phone
+1 (800) 983-4587
Email
GMI@bmrn.com
First Name & Middle Initial & Last Name or Official Title & Degree
MedInfo
Phone
+1 (800) 983-4587
Email
medinfo@bmr n.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Medinfo Medinfo
Organizational Affiliation
BioMarin Pharmaceutical
Official's Role
Study Director
Facility Information:
Facility Name
Medico Hematologo
City
Mendoza
Country
Argentina
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
medinfo
Email
GMI@bmrn.com
12. IPD Sharing Statement
Plan to Share IPD
No
Learn more about this trial
A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
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