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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05580692




Registration number
NCT05580692
Ethics application status
Date submitted
16/09/2022
Date registered
14/10/2022
Date last updated
19/08/2024

Titles & IDs
Public title
A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
Scientific 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
Secondary ID [1] 0 0
270-702
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hemophilia A 0 0
Condition category
Condition code
Blood 0 0 0 0
Clotting disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Surgery - Biospecimen Collection

Experimental: Patient Cohort - All Patients are within the same arm


Treatment: Surgery: Biospecimen Collection
Biospecimen Sample Collection
Intervention code [1] 0 0
Treatment: Surgery
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
To qunatify Seroprevalence of antibodies to selected AAV serotypes
Timepoint [1] 0 0
52 weeks
Secondary outcome [1] 0 0
To describe and characterize selected AAV serotypes antibody responses in adult subjects with Hemophilia A
Timepoint [1] 0 0
52 weeks

Eligibility
Key inclusion criteria
* Adult Subjects with Hemophilia A
Minimum age
18 Years
Maximum age
No limit
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Subject previously treated with AAV vector gene therapy

Study design
Purpose of the study
Other
Allocation to intervention
Not applicable
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Not applicable
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Stopped early
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
14/03/2023
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
Actual
31/07/2024
Sample size
Target
Accrual to date
Final
321
Recruitment in Australia
Recruitment state(s)
Recruitment hospital [1] 0 0
Royal Prince Alfred Hospital - Camperdown
Recruitment hospital [2] 0 0
Haemophilia and Haemostasis Centre Level 1 Cancer Centre South Metropolitan Health Service Fiona Stanley Hospital - Murdoch
Recruitment postcode(s) [1] 0 0
2050 - Camperdown
Recruitment postcode(s) [2] 0 0
6150 - Murdoch
Recruitment outside Australia
Country [1] 0 0
Argentina
State/province [1] 0 0
Mendoza
Country [2] 0 0
Argentina
State/province [2] 0 0
Corrientes
Country [3] 0 0
Argentina
State/province [3] 0 0
Rosario
Country [4] 0 0
Austria
State/province [4] 0 0
Wien
Country [5] 0 0
Saudi Arabia
State/province [5] 0 0
Riyadh
Country [6] 0 0
Sweden
State/province [6] 0 0
Malmö
Country [7] 0 0
Sweden
State/province [7] 0 0
Solna
Country [8] 0 0
Turkey
State/province [8] 0 0
Adana
Country [9] 0 0
Turkey
State/province [9] 0 0
Ankara
Country [10] 0 0
Turkey
State/province [10] 0 0
Gaziantep
Country [11] 0 0
Turkey
State/province [11] 0 0
Istanbul
Country [12] 0 0
Turkey
State/province [12] 0 0
Izmir
Country [13] 0 0
Turkey
State/province [13] 0 0
Samsun
Country [14] 0 0
United Arab Emirates
State/province [14] 0 0
Abu Dhabi

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
BioMarin Pharmaceutical
Address
Country

Ethics approval
Ethics application status

Summary
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
Trial website
https://clinicaltrials.gov/study/NCT05580692
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Medinfo Medinfo
Address 0 0
BioMarin Pharmaceutical
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT05580692