CClinicalTrials.gg
TerminatedNCT05580692Updated Aug 19, 2024

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

An interventional study of Biospecimen Collection in Hemophilia A, sponsored by BioMarin Pharmaceutical. Terminated at 18 sites in 7 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-08-19.

Sponsored by BioMarin Pharmaceutical · Not applicable, Interventional, and Other

Why this study was terminated
Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved.
Phase
Not applicable
Study type
Interventional
Enrollment
321
Allocation
Not applicable
Ages
18 Years and older
Sex
All
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Study 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

Read the 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

02

Conditions studied

  • Hemophilia A

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03

In context

Hemophilia A

866 studies on the registry are indexed under Hemophilia A; 137 are open to participants now.

This study's enrollment of 321 is above the median of 28 across 512 interventional studies indexed under Hemophilia A.

Browse Hemophilia A studies →

Lead sponsor

BioMarin Pharmaceutical is the lead sponsor of 110 studies on the registry; 13 are open to participants now.

Of its 17 completed or terminated interventional studies of FDA-regulated products, 12 (71%) have results posted.

Counted across the registry records on this site, refreshed daily.

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Who can participate

Ages eligible
18 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Inclusion criteria

  • Adult Subjects with Hemophilia A

Exclusion criteria

Exclusion Criteria:

  • Subject previously treated with AAV vector gene therapy
05

Study design

Phase
Not applicable
Primary purpose
Other
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
321 participants (actual)

Study arms

  • Experimental
    Patient Cohort

    All Patients are within the same arm

    Procedure: Biospecimen Collection

Interventions

  • ProcedureBiospecimen Collection

    Biospecimen Sample Collection

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What researchers measure

Primary outcomes

  1. To qunatify Seroprevalence of antibodies to selected AAV serotypes

    Number of patients with detectable antibodies to selected AAV serotypes

    Time frame: 52 weeks

Secondary outcomes

  1. 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

    Time frame: 52 weeks

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Study locations

18 sites
  • Instituto Hematologia Arbesu
    Godoy Cruz, Mendoza M5504FKD, Argentina
  • Hospital Ramon Jose Vidal
    Corrientes, W3410 FND, Argentina
  • Medico Hematologo
    Mendoza, Argentina
  • Instituto de Hematologia y Medicina Clinica Dr. RUben Davoli
    Rosario, S2000 CFK, Argentina
  • Royal Prince Alfred Hospital
    Camperdown, 2050, Australia
  • Haemophilia and Haemostasis Centre Level 1 Cancer Centre South Metropolitan Health Service Fiona Stanley Hospital
    Murdoch, 6150, Australia
  • Medical University of Vienna
    Wien, 1090, Austria
  • King Faisal Specialist Hospital & Research Center
    Riyadh, 11211, Saudi Arabia
  • King Saud University Medical City King Saud University
    Riyadh, 12372, Saudi Arabia
  • Skane University Hospital
    Malmö, 205 02, Sweden
  • ME Centrum för kliniska cancerstudier - House H3
    Solna, SE-171 76, Sweden
  • Acibadem Adana Hospital
    Adana, 01130, Turkey
  • Hacettepe University
    Ankara, 06100, Turkey
  • Gaziantep University Medical Faculty Sahinbey Educational Research Hospital
    Gaziantep, 27310, Turkey
  • Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty
    Istanbul, 34098, Turkey
  • Ege University Medical Faculty
    İzmir, 35040, Turkey
  • Ondokuz Mayis Univ. Med. Fac.
    Samsun, 55270, Turkey
  • Sheikh Shakhbout Medical City
    Abu Dhabi, 11393, United Arab Emirates
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References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Aug 19, 2024, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
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Registry details

Key details

Study ID
NCT05580692
Lead sponsor
BioMarin Pharmaceutical
Responsible party
Sponsor
First posted
Oct 14, 2022
Start date
Mar 14, 2023
Primary completion
Jul 31, 2024
Completion
Jul 31, 2024
Last update
Aug 19, 2024

Study contacts

Medinfo Medinfo
study director · BioMarin Pharmaceutical

Oversight

Data monitoring committee
No
FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is terminated, as verified in Aug 2024. You cannot join it, but the record below documents what was studied.

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