Hemophilia ASAAVYNCT04560933
A Study to Evaluate Seroprevalence and Seroconversion of Antibodies to Adeno-Associated Virus (AAV) in Patients With Hemophilia A
An observational study of Hemophilia A, run by BioMarin Pharmaceutical at 1 site in United States.
Trial summary
To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A 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?
- Blood sample collection
- Type of treatment
- Surgery or procedure
- How many people
- 186 participants
- Where
- 1 siteUnited States
- Study dates
- 25 Aug 2020to 20 Oct 2023
- Phase
- Observational
- 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
- Adult, Older adult
Study population: Subjects diagnosed with Hemophilia A
Who may be able to join
- Subjects diagnosed with Hemophilia A
- Subjects aged 18years or over at time of entry
Who cannot join
- Currently participating in an interventional study of any investigational product, device or procedure.
- Subjects who have been previously treated with AAV vector gene therapy
Trial locations and contacts
1 site in 1 country.
About this study
Official title: A Prospective, Observational Study Evaluating Seroprevalence and Rate of Seroconversion of Antibodies Against Adeno-associated Virus (AAV) Serotypes and Exploratory Vectors in Subjects With Hemophilia A in the United States
To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A
Read the detailed description
This is a single-center, decentralized, patient-centered, prospective, observational study utilizing biospecimen samples collected from hemophilia A subjects across the United States to evaluate and characterize seroprevalence and the rate of seroconversion of antibodies against AAV serotypes and exploratory vectors, and to investigate the associated factors that may influence the vector titers.
Relevant medical findings will also be collected from the subject, as well as symptoms related to hemophilia A. The collection of medical history may include major illnesses, diagnoses, and surgeries.
- Conditions
- Hemophilia A
- Being tested
- Blood sample 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.
Blood sample collectionProcedure or surgery
Blood sample collection
What is being measured
The primary outcome is the main question the study is designed to answer.
Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A
To assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A at Baseline
Measured: Baseline
Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A
Measured: Change from baseline through Week 12
Secondary outcomes (2)
To describe and characterize AAV vector titer values in subjects with Hemophilia A
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Measured: Change from baseline through Week 12
To describe and characterize AAV vector titer values in subjects with Hemophilia A
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Measured: Change from baseline through Week 24
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2020
- Study start25 Aug 2020
- Primary completion20 Sep 2023
- Study completion20 Oct 2023
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
- Observational
- Observational model
- Ecologic or community
- Time perspective
- Prospective
- Enrolment
- 186 participants (actual)
Sponsor and investigators
- Lead sponsor
- BioMarin Pharmaceutical
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Stephen Flach, MD, Principal investigator, Covance
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04560933
- Sponsor's protocol ID
- 270-701
- Acronym
- SAAVY
- Status last verified
- Apr 2024
- First submitted
- 15 Sep 2020
- Last updated
- 24 Apr 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 24 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Prospective, Observational Study Evaluating Seroprevalence and Rate of Seroconversion of Antibodies Against Adeno-associated Virus (AAV) Serotypes and Exploratory Vectors in Subjects With Hemophilia A in the United States
- ClinicalTrials.gov ID
- NCT04560933
- Acronym / sponsor ID
- SAAVY / 270-701
- Conditions
- Hemophilia A
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 186 participants (actual)
- Lead sponsor
- BioMarin PharmaceuticalIndustry (pharma, biotech, devices)
- Study start
- 25 Aug 2020 (actual)
- Primary completion
- 20 Sep 2023 (actual)
- Study completion
- 20 Oct 2023 (actual)
- First posted
- 23 Sep 2020 (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.
To quantify the seroprevalence of antibodies to AAV5, AAV6, and AAV8 and the seroconversion rate over varying follow-up intervals in subjects with hemophilia A
Detailed description
This is a single-center, decentralized, patient-centered, prospective, observational study utilizing biospecimen samples collected from hemophilia A subjects across the United States to evaluate and characterize seroprevalence and the rate of seroconversion of antibodies against AAV serotypes and exploratory vectors, and to investigate the associated factors that may influence the vector titers.
Relevant medical findings will also be collected from the subject, as well as symptoms related to hemophilia A. The collection of medical history may include major illnesses, diagnoses, and surgeries.
Study design
- Observational model
- Ecologic or community
- Time perspective
- Prospective
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Age groups
- Adult, Older adult
- Study population
- Subjects diagnosed with Hemophilia A
Key inclusion criteria (2 listed)
- Subjects diagnosed with Hemophilia A
- Subjects aged 18years or over at time of entry
Key exclusion criteria (2 listed)
- Currently participating in an interventional study of any investigational product, device or procedure.
- Subjects who have been 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 |
|---|---|---|
| Blood sample collectionBlood sample collection | Procedure or surgery | – |
Primary outcome
Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A
Time frame: Baseline
To assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A at Baseline
Assess the rate of Seroconversion of antibodies to AAV vectors in patients with hemophilia A
Time frame: Change from baseline through Week 12
2 secondary outcomes
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Time frame: Change from baseline through Week 12
To describe and characterize AAV vector titer values in subjects with Hemophilia A
Time frame: Change from baseline through Week 24
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 24 Apr 2024.
- Individual participant dataSharing planned: No
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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.
Stephen Flach, MD principal investigator
Covance
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A, run by BioMarin Pharmaceutical at 1 site in United States.
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 2 inclusion and 2 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: finished, as of the sponsor's last update on 24 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. 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 is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
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?
Assess the Seroprevalence of antibodies to AAV vectors in patients with hemophilia A (time frame: Baseline). The study lists 2 primary outcomes in all. 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 24 Apr 2024.