Hemophilia ANCT05580692
A Prospective Study Evaluating Seroprevalence and Seroconversion of Antibodies Against Adeno-associated Virus (AAV)
A study testing Biospecimen Collection in people with Hemophilia A, run by BioMarin Pharmaceutical at 18 sites in 7 countries.
Trial 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 more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Hemophilia A
- What is being tested?
- Biospecimen Collection
- Type of treatment
- Surgery or procedure
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 321 participants
- Where
- 18 sitesin 7 countries
- Study dates
- 14 Mar 2023to 31 Jul 2024
- Phase
- Interventional
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Adult Subjects with Hemophilia A
Who cannot join
- Subject previously treated with AAV vector gene therapy
Trial locations and contacts
18 sites in 7 countries.
18 sites in 7 countries
Turkey (Türkiye)6 sites
Acibadem Adana Hospital
Adana, 01130
Hacettepe University
Ankara, 06100
Gaziantep University Medical Faculty Sahinbey Educational Research Hospital
Gaziantep, 27310
Istanbul University Cerrahpasa - Cerrahpasa Medical Faculty
Istanbul, 34098
Ege University Medical Faculty
Izmir, 35040
Ondokuz Mayis Univ. Med. Fac.
Samsun, 55270
Argentina4 sites
Hospital Ramon Jose Vidal
Corrientes, W3410 FND
Instituto Hematologia Arbesu
Godoy Cruz, Mendoza Province, M5504FKD
Medico Hematologo
Mendoza
Instituto de Hematologia y Medicina Clinica Dr. RUben Davoli
Rosario, S2000 CFK
Australia2 sites
Royal Prince Alfred Hospital
Camperdown, 2050
Haemophilia and Haemostasis Centre Level 1 Cancer Centre South Metropolitan Health Service Fiona Stanley Hospital
Murdoch, 6150
Saudi Arabia2 sites
King Faisal Specialist Hospital & Research Center
Riyadh, 11211
King Saud University Medical City King Saud University
Riyadh, 12372
Sweden2 sites
Skane University Hospital
Malmö, 205 02
ME Centrum för kliniska cancerstudier - House H3
Solna, SE-171 76
United Arab Emirates1 site
Sheikh Shakhbout Medical City
Abu Dhabi, 11393
About this study
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
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
- Conditions
- Hemophilia A
- Being tested
- Biospecimen Collection
- Medical subject headings
- Hemophilia A
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Biospecimen CollectionProcedure or surgery
Biospecimen Sample Collection
| Study group | Role | What they receive |
|---|---|---|
| Patient Cohort | Experimental | All Patients are within the same arm Receives: Procedure: Biospecimen Collection |
What is being measured
The primary outcome is the main question the study is designed to answer.
To qunatify Seroprevalence of antibodies to selected AAV serotypes
Number of patients with detectable antibodies to selected AAV serotypes
Measured: 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
Measured: 52 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Oct 2022
- Study start14 Mar 2023
- Primary completion31 Jul 2024
- Study completion31 Jul 2024
Why it stopped: Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved.
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Hemophilia AStudies of the same condition, open and under way.
- More trials run by BioMarin PharmaceuticalThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 321 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Medinfo Medinfo, Study director, BioMarin Pharmaceutical
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05580692
- Sponsor's protocol ID
- 270-702
- Status last verified
- Aug 2024
- First submitted
- 16 Sep 2022
- Last updated
- 19 Aug 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 19 Aug 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT05580692
- Acronym / sponsor ID
- 270-702
- Conditions
- Hemophilia A
- Study type
- Interventional
- Recruitment status
- TerminatedReason stopped: Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved.
- Enrolment
- 321 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 14 Mar 2023 (actual)
- Primary completion
- 31 Jul 2024 (actual)
- Study completion
- 31 Jul 2024 (actual)
- First posted
- 14 Oct 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patient Cohort | Experimental | Procedure: Biospecimen CollectionAll Patients are within the same arm |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- Adult Subjects with Hemophilia A
Key exclusion criteria (1 listed)
- Subject previously treated with AAV vector gene therapy
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Biospecimen CollectionBiospecimen Sample Collection | Procedure or surgery | Patient Cohort |
Primary outcome
To qunatify Seroprevalence of antibodies to selected AAV serotypes
Time frame: 52 weeks
Number of patients with detectable antibodies to selected AAV serotypes
1 secondary outcome
To describe and characterize selected AAV serotypes antibody responses in adult subjects with Hemophilia A
Time frame: 52 weeks
Results and safety
The study was stopped early (reason given: Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved.). Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 19 Aug 2024.
- Individual participant dataSharing planned: No
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A Study to Learn About the Study Medicine -Hympavzi in Congenital Hemophilia Patients Without Inhibitors in Japan.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Medinfo Medinfo study director
BioMarin Pharmaceutical
18 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing Biospecimen Collection in people with Hemophilia A, run by BioMarin Pharmaceutical at 18 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: stopped early, as of the sponsor's last update on 19 Aug 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 18 sites in Turkey (Türkiye), Argentina, Australia, Saudi Arabia, Sweden and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study was stopped early (reason given: Sponsor decision to terminate; sufficient sample size for statistical analysis has been achieved.). Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To qunatify Seroprevalence of antibodies to selected AAV serotypes (time frame: 52 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 19 Aug 2024.