Hemophilia ANCT03185897
Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia
An observational study of Hemophilia A and Hemophilia B, run by Baxalta now part of Shire at 23 sites in 6 countries.
Trial summary
Assess the seroprevalence of neutralizing antibodies (NAb) to AAV in adults with severe hemophilia A (coagulation factor VIII [FVIII] <1%) or moderately severe to severe hemophilia B (coagulation factor IX [FIX] ≤2%). Read more about this study
- Who is it for?
- Ages 18 to 75 years, men only
- Conditions the study is for
- Hemophilia A, Hemophilia B
- What is being tested?
- Non-treatment, seroprevalence
- How many people
- 242 participants
- Where
- 23 sitesin 6 countries
- Study dates
- 14 Jun 2017to 17 Mar 2021
- 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 to 75 years
- Men only
- No healthy volunteers
- Adult, Older adult
Study population: Hemophilia A and hemophilia B patients who receive treatment at Hemophilia Treatment Centers.
Who may be able to join
- Participant is male between 18 and 75 years old at the time of screening.
- Established severe hemophilia A (plasma Factor VIII (FVIII) activity <1%) or B (plasma Factor IX (FIX) activity ≤2%).
- Provision of signed informed consent form (ICF).
- Participant is willing and able to comply with the requirements of the protocol.
Who cannot join
- Bleeding disorder(s) other than hemophilia A or B.
- Personal laboratory evidence of having developed inhibitors to FVIII or FIX protein at any time (≥0.6 Bethesda Units [BU] on any single test).
- Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy.
- Received systemic antiviral and/or interferon therapy within 30 days of blood draw, with the exception of treatment for human immunodeficiency virus (HIV).
- Currently receiving ribavirin and/or interferon based therapy for active hepatitis C virus (HCV).
- Received immunoglobulin therapy or plasma infusion within 120 days of the blood draw.
Show all 9Show fewer
- Has a known immune deficiency other than HIV.
- Has lymphocyte or plasma cell malignancies.
- Participant is a family member or employee of the investigator.
Trial locations and contacts
23 sites in 6 countries.
23 sites in 6 countries
United States7 sites
University of Colorado Hemophilia & Thrombosis Center
Aurora, Colorado, 80045
Medical University of South Carolina (MUSC)
Charleston, South Carolina, 29425
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Michigan State University
East Lansing, Michigan, 48824
Gulf States Hemophilia and Thrombophilia Center
Houston, Texas, 77030
Orthopaedic Hemophilia Treatment Center
Los Angeles, California, 90007
University of Washington
Seattle, Washington, 98104
France6 sites
Hôpital Morvan
Brest, 29609
Groupement Hospitalier Est- Hôpital Louis Pradel
Bron, 69677
Groupement Hospitalier Sud - Hôpital Bicêtre
Le Kremlin-Bicêtre, 94275
Hopital Jeanne de Flandre - CHU Lille
Lille, 59037
Hôpital de la Timone
Marseille, 13385
CHU de Nantes Site Hotel Dieu
Nantes, 44093
Italy4 sites
Presidio Ospedaliero di Castelfranco Veneto
Castelfranco Veneto, 31033
Azienda Ospedaliera Universitaria Careggi
Florence, 50134
Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico
Milan, 20122
Ospedale San Bortolo di Vicenza
Vicenza, 36100
Spain3 sites
Hospital Universitario La Paz
Madrid, 28046
Hospital Regional Universitario de Malaga
Málaga, 29010
Hospital Universitario de Salamanca
Salamanca, 37007
Germany2 sites
Vivantes Klinikum im Friedrichshain
Berlin, 10249
Klinikum der Johann Wolfgang Goethe-Universitaet
Frankfurt, 60590
About this study
Official title: A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults With Hemophilia
Assess the seroprevalence of neutralizing antibodies (NAb) to AAV in adults with severe hemophilia A (coagulation factor VIII [FVIII] <1%) or moderately severe to severe hemophilia B (coagulation factor IX [FIX] ≤2%).
- Conditions
- Hemophilia A, Hemophilia B
- Being tested
- Non-treatment, seroprevalence
- Medical subject headings
- Hemophilia A, Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Non-treatment, seroprevalenceOther
Non-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV)
| Study group | Role | What they receive |
|---|---|---|
| Hemophilia A | Participants with hemophilia A Receives: Other: Non-treatment, seroprevalence | |
| Hemophilia B | Participants with hemophilia B Receives: Other: Non-treatment, seroprevalence |
What is being measured
The primary outcome is the main question the study is designed to answer.
Prevalence of neutralizing antibodies (NAb) - Baseline visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Measured: Baseline visit
Prevalence of neutralizing antibodies (NAb) - Year 1 Visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Measured: Year 1 visit
Prevalence of neutralizing antibodies (NAb) - Year 2 Visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Measured: Year 2 visit
Prevalence of neutralizing antibodies (NAb) - Year 3 Visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Measured: Year 3 visit
Secondary outcomes (3)
Prevalence of NAb to AAV including AAV2 and AAV8
Prevalence of neutralizing antibodies (NAb) to adeno-associated virus (AAV) including AAV2 and AAV8
Measured: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
Prevalence of binding antibodies to AAV, including AAV8 and AAV2
Prevalence of binding antibodies to adeno-associated virus (AAV), including AAV8 and AAV2
Measured: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
T-cell mediated immune response to AAV8
T-cell mediated immune response to AAV8
Measured: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted14 Jun 2017
- Study start14 Jun 2017
- Primary completion17 Mar 2021
- Study completion17 Mar 2021
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.
- 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 242 participants (actual)
Sponsor and investigators
- Lead sponsor
- Baxalta now part of Shire
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Study Director, Study director, Shire
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03185897
- Sponsor's protocol ID
- 201601
- Status last verified
- Oct 2021
- First submitted
- 31 May 2017
- Last updated
- 25 Oct 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Yes: Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
Study at a glance
From the registry record, last updated by the sponsor on 25 Oct 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Global Epidemiologic Study to Determine the Prevalence of Neutralizing Antibodies and Related Adaptive Immune Responses to Adeno-Associated Virus (AAV) in Adults With Hemophilia
- ClinicalTrials.gov ID
- NCT03185897
- Acronym / sponsor ID
- 201601
- Conditions
- Hemophilia A, Hemophilia B
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 242 participants (actual)
- Lead sponsor
- Baxalta now part of ShireIndustry (pharma, biotech, devices)
- Study start
- 14 Jun 2017 (actual)
- Primary completion
- 17 Mar 2021 (actual)
- Study completion
- 17 Mar 2021 (actual)
- First posted
- 14 Jun 2017 (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.
Assess the seroprevalence of neutralizing antibodies (NAb) to AAV in adults with severe hemophilia A (coagulation factor VIII [FVIII] <1%) or moderately severe to severe hemophilia B (coagulation factor IX [FIX] ≤2%).
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Hemophilia A | – | Other: Non-treatment, seroprevalenceParticipants with hemophilia A |
| Hemophilia B | – | Other: Non-treatment, seroprevalenceParticipants with hemophilia B |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
- Study population
- Hemophilia A and hemophilia B patients who receive treatment at Hemophilia Treatment Centers.
Key inclusion criteria (4 listed)
- Participant is male between 18 and 75 years old at the time of screening.
- Established severe hemophilia A (plasma Factor VIII (FVIII) activity <1%) or B (plasma Factor IX (FIX) activity ≤2%).
- Provision of signed informed consent form (ICF).
- Participant is willing and able to comply with the requirements of the protocol.
Key exclusion criteria (9 listed)
- Bleeding disorder(s) other than hemophilia A or B.
- Personal laboratory evidence of having developed inhibitors to FVIII or FIX protein at any time (≥0.6 Bethesda Units [BU] on any single test).
- Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy.
- Received systemic antiviral and/or interferon therapy within 30 days of blood draw, with the exception of treatment for human immunodeficiency virus (HIV).
- Currently receiving ribavirin and/or interferon based therapy for active hepatitis C virus (HCV).
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 |
|---|---|---|
| Non-treatment, seroprevalenceNon-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV) | Other | Hemophilia A, Hemophilia B |
Primary outcome
Prevalence of neutralizing antibodies (NAb) - Baseline visit
Time frame: Baseline visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Prevalence of neutralizing antibodies (NAb) - Year 1 Visit
Time frame: Year 1 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Prevalence of neutralizing antibodies (NAb) - Year 2 Visit
Time frame: Year 2 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Prevalence of neutralizing antibodies (NAb) - Year 3 Visit
Time frame: Year 3 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
3 secondary outcomes
Prevalence of NAb to AAV including AAV2 and AAV8
Time frame: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
Prevalence of binding antibodies to AAV, including AAV8 and AAV2
Time frame: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
T-cell mediated immune response to AAV8
Time frame: Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit
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 25 Oct 2021.
- To obtain more information on the study, click here/on this linkLink provided by the sponsor.
- Individual participant dataSharing planned: Yes. Takeda provides access to the de-identified individual participant data (IPD) for eligible studies to aid qualified researchers in addressing legitimate scientific objectives (Takeda's data sharing commitment is available on https://clinicaltrials.takeda.com/takedas-commitment?commitment=5). These IPDs will be provided in a secure research environment following approval of a data sharing request, and under the terms of a data sharing agreement.
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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.
Study Director study director
Shire
23 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Hemophilia A and Hemophilia B, run by Baxalta now part of Shire at 23 sites in 6 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 4 inclusion and 9 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 25 Oct 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 23 sites in United States, France, Italy, Spain, Germany and 1 more country. 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?
Prevalence of neutralizing antibodies (NAb) - Baseline visit (time frame: Baseline visit). The study lists 4 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 25 Oct 2021.