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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03185897
Recruitment Status : Active, not recruiting
First Posted : June 14, 2017
Last Update Posted : November 29, 2018
Sponsor:
Information provided by (Responsible Party):
Shire ( Baxalta now part of Shire )

Brief 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%).

Condition or disease Intervention/treatment
Hemophilia A Hemophilia B Other: Non-treatment, seroprevalence

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Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Cohort
Time Perspective: Prospective
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
Actual Study Start Date : June 14, 2017
Estimated Primary Completion Date : April 30, 2022
Estimated Study Completion Date : April 30, 2022

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Hemophilia

Group/Cohort Intervention/treatment
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)




Primary Outcome Measures :
  1. Prevalence of neutralizing antibodies (NAb) - Baseline visit [ Time Frame: Baseline visit ]
    Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes

  2. Prevalence of neutralizing antibodies (NAb) - Year 1 Visit [ Time Frame: Year 1 visit ]
    Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes

  3. Prevalence of neutralizing antibodies (NAb) - Year 2 Visit [ Time Frame: Year 2 visit ]
    Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes

  4. Prevalence of neutralizing antibodies (NAb) - Year 3 Visit [ Time Frame: Year 3 visit ]
    Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes


Secondary Outcome Measures :
  1. 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

  2. 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

  3. 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



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Ages Eligible for Study:   18 Years to 75 Years   (Adult, Older Adult)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Hemophilia A and hemophilia B patients who receive treatment at Hemophilia Treatment Centers.
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03185897


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

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Responsible Party: Baxalta now part of Shire
ClinicalTrials.gov Identifier: NCT03185897     History of Changes
Other Study ID Numbers: 201601
First Posted: June 14, 2017    Key Record Dates
Last Update Posted: November 29, 2018
Last Verified: November 2018

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
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Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked