Global Epidemiologic Study of Preexisting Immunity to AAV in Adults With Severe Hemophilia

October 22, 2021 updated by: Baxalta now part of Shire

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%).

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
242

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Vienna, Austria, 1090
        • AKH - Medizinische Universitat Wien
  • France
      • Brest Cedex, France, 29609
        • Hopital Morvan
      • Bron cedex, France, 69677
        • Groupement Hospitalier Est- Hôpital Louis Pradel
      • Le Kremlin Bicêtre cedex, France, 94275
        • Groupement Hospitalier Sud - Hôpital Bicêtre
      • Lille Cedex, France, 59037
        • Hopital Jeanne de Flandre - CHU Lille
      • Marseille Cedex 05, France, 13385
        • Hopital De La Timone
      • Nantes Cedex 1, France, 44093
        • CHU de Nantes Site Hotel Dieu
  • Germany
      • Berlin, Germany, 10249
        • Vivantes Klinikum im Friedrichshain
      • Frankfurt, Germany, 60590
        • Klinikum Der Johann Wolfgang Goethe-Universitaet
  • Italy
      • Castelfranco Veneto, Italy, 31033
        • Presidio Ospedaliero di Castelfranco Veneto
      • Firenze, Italy, 50134
        • Azienda Ospedaliera Universitaria Careggi
      • Milano, Italy, 20122
        • Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
      • Vicenza, Italy, 36100
        • Ospedale San Bortolo di Vicenza
  • Spain
      • Madrid, Spain, 28046
        • Hospital Universitario La Paz
      • Malaga, Spain, 29010
        • Hospital Regional Universitario de Málaga
      • Salamanca, Spain, 37007
        • Hospital Universitario de Salamanca
  • United States
    • California
      • Los Angeles, California, United States, 90007
        • Orthopaedic Hemophilia Treatment Center
    • Colorado
      • Aurora, Colorado, United States, 80045
        • University of Colorado Hemophilia & Thrombosis Center
    • Michigan
      • East Lansing, Michigan, United States, 48824
        • Michigan State University
    • Ohio
      • Cincinnati, Ohio, United States, 45229
        • Cincinnati Children's Hospital Medical Center
    • South Carolina
      • Charleston, South Carolina, United States, 29425
        • Medical University of South Carolina (MUSC)
    • Texas
      • Houston, Texas, United States, 77030
        • Gulf States Hemophilia and Thrombophilia Center
    • Washington
      • Seattle, Washington, United States, 98104
        • University of Washington

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

14 years to 71 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Sampling Method

Non-Probability Sample

Study Population

Hemophilia A and hemophilia B patients who receive treatment at Hemophilia Treatment Centers.

Description

Inclusion Criteria:

  1. Participant is male between 18 and 75 years old at the time of screening.
  2. Established severe hemophilia A (plasma Factor VIII (FVIII) activity <1%) or B (plasma Factor IX (FIX) activity ≤2%).
  3. Provision of signed informed consent form (ICF).
  4. Participant is willing and able to comply with the requirements of the protocol.

Exclusion Criteria:

  1. Bleeding disorder(s) other than hemophilia A or B.
  2. Personal laboratory evidence of having developed inhibitors to FVIII or FIX protein at any time (≥0.6 Bethesda Units [BU] on any single test).
  3. Currently receiving systemic immunosuppressive therapy, cytotoxic chemotherapy, immunoglobulin therapy, or monoclonal antibody therapy.
  4. Received systemic antiviral and/or interferon therapy within 30 days of blood draw, with the exception of treatment for human immunodeficiency virus (HIV).
  5. Currently receiving ribavirin and/or interferon based therapy for active hepatitis C virus (HCV).
  6. Received immunoglobulin therapy or plasma infusion within 120 days of the blood draw.
  7. Has a known immune deficiency other than HIV.
  8. Has lymphocyte or plasma cell malignancies.
  9. Participant is a family member or employee of the investigator.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

Group / Cohort

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Hemophilia A
Participants with hemophilia A
Other: Non-treatment, seroprevalence
Non-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV)
Hemophilia B
Participants with hemophilia B
Other: Non-treatment, seroprevalence
Non-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV)

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Prevalence of neutralizing antibodies (NAb) - Baseline visit
Time Frame: Baseline visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Baseline visit
Prevalence of neutralizing antibodies (NAb) - Year 1 Visit
Time Frame: Year 1 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Year 1 visit
Prevalence of neutralizing antibodies (NAb) - Year 2 Visit
Time Frame: Year 2 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Year 2 visit
Prevalence of neutralizing antibodies (NAb) - Year 3 Visit
Time Frame: Year 3 visit
Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes
Year 3 visit

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
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 neutralizing antibodies (NAb) to adeno-associated virus (AAV) including AAV2 and AAV8
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
Prevalence of binding antibodies to adeno-associated virus (AAV), including AAV8 and AAV2
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
T-cell mediated immune response to AAV8
Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Baxalta now part of Shire

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
June 14, 2017

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
March 17, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
March 17, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
May 31, 2017

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 12, 2017

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
June 14, 2017

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
October 25, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 22, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
October 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • 201601

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

Yes

IPD Plan Description

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.

IPD Sharing Access Criteria

IPD from eligible studies will be shared with qualified researchers according to the criteria and process described on https://vivli.org/ourmember/takeda/. For approved requests, the researchers will be provided access to anonymized data (to respect patient privacy in line with applicable laws and regulations) and with information necessary to address the research objectives under the terms of a data sharing agreement.

IPD Sharing Supporting Information Type

  • Study Protocol
  • Statistical Analysis Plan (SAP)
  • Informed Consent Form (ICF)
  • Clinical Study Report (CSR)

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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Dietary Supplements

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