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

NCT ID: NCT03185897

Last Updated: 2021-10-25

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

242 participants

Study Classification

OBSERVATIONAL

Study Start Date

2017-06-14

Study Completion Date

2021-03-17

Brief Summary

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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%).

Detailed Description

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Conditions

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Hemophilia A Hemophilia B

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Study Groups

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Hemophilia A

Participants with hemophilia A

Non-treatment, seroprevalence

Intervention Type OTHER

Non-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV)

Hemophilia B

Participants with hemophilia B

Non-treatment, seroprevalence

Intervention Type OTHER

Non-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV)

Interventions

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Non-treatment, seroprevalence

Non-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV)

Intervention Type OTHER

Eligibility Criteria

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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.
Minimum Eligible Age

18 Years

Maximum Eligible Age

75 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

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Baxalta now part of Shire

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Study Director

Role: STUDY_DIRECTOR

Shire

Locations

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Orthopaedic Hemophilia Treatment Center

Los Angeles, California, United States

Site Status

University of Colorado Hemophilia & Thrombosis Center

Aurora, Colorado, United States

Site Status

Michigan State University

East Lansing, Michigan, United States

Site Status

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, United States

Site Status

Medical University of South Carolina (MUSC)

Charleston, South Carolina, United States

Site Status

Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, United States

Site Status

University of Washington

Seattle, Washington, United States

Site Status

AKH - Medizinische Universität Wien

Vienna, , Austria

Site Status

Hôpital Morvan

Brest, , France

Site Status

Groupement Hospitalier Est- Hôpital Louis Pradel

Bron, , France

Site Status

Groupement Hospitalier Sud - Hôpital Bicêtre

Le Kremlin-Bicêtre, , France

Site Status

Hopital Jeanne de Flandre - CHU Lille

Lille, , France

Site Status

Hôpital de la Timone

Marseille, , France

Site Status

CHU de Nantes Site Hotel Dieu

Nantes, , France

Site Status

Vivantes Klinikum im Friedrichshain

Berlin, , Germany

Site Status

Klinikum der Johann Wolfgang Goethe-Universitaet

Frankfurt, , Germany

Site Status

Presidio Ospedaliero di Castelfranco Veneto

Castelfranco Veneto, , Italy

Site Status

Azienda Ospedaliera Universitaria Careggi

Florence, , Italy

Site Status

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, , Italy

Site Status

Ospedale San Bortolo di Vicenza

Vicenza, , Italy

Site Status

Hospital Universitario La Paz

Madrid, , Spain

Site Status

Hospital Regional Universitario de Malaga

Málaga, , Spain

Site Status

Hospital Universitario de Salamanca

Salamanca, , Spain

Site Status

Countries

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United States Austria France Germany Italy Spain

Related Links

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https://clinicaltrials.takeda.com/study-detail/5f6b5fc54db2bf003ab45d07

To obtain more information on the study, click here/on this link

Other Identifiers

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201601

Identifier Type: -

Identifier Source: org_study_id