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

Sponsor
Baxalta now part of Shire (Industry)
Overall Status
Completed
CT.gov ID
NCT03185897
Collaborator
(none)
242
Enrollment
23
Locations
45.1
Actual Duration (Months)
10.5
Patients Per Site
0.2
Patients Per Site Per Month

Study Details

Study Description

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 Phase
  • Other: Non-treatment, seroprevalence

Study Design

Study Type:
Observational
Actual Enrollment :
242 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 :
Jun 14, 2017
Actual Primary Completion Date :
Mar 17, 2021
Actual Study Completion Date :
Mar 17, 2021

Arms and Interventions

Arm 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)

Outcome Measures

Primary Outcome Measures

  1. Prevalence of neutralizing antibodies (NAb) - Baseline visit [Baseline visit]

    Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes

  2. Prevalence of neutralizing antibodies (NAb) - Year 1 Visit [Year 1 visit]

    Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes

  3. Prevalence of neutralizing antibodies (NAb) - Year 2 Visit [Year 2 visit]

    Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes

  4. Prevalence of neutralizing antibodies (NAb) - Year 3 Visit [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 [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 [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 [Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit]

    T-cell mediated immune response to AAV8

Eligibility Criteria

Criteria

Ages Eligible for Study:
18 Years to 75 Years
Sexes Eligible for Study:
Male
Accepts Healthy Volunteers:
No
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.

Contacts and Locations

Locations

Site City State Country Postal Code
1 Orthopaedic Hemophilia Treatment Center Los Angeles California United States 90007
2 University of Colorado Hemophilia & Thrombosis Center Aurora Colorado United States 80045
3 Michigan State University East Lansing Michigan United States 48824
4 Cincinnati Children's Hospital Medical Center Cincinnati Ohio United States 45229
5 Medical University of South Carolina (MUSC) Charleston South Carolina United States 29425
6 Gulf States Hemophilia and Thrombophilia Center Houston Texas United States 77030
7 University of Washington Seattle Washington United States 98104
8 AKH - Medizinische Universität Wien Vienna Austria 1090
9 Hôpital Morvan Brest Cedex France 29609
10 Groupement Hospitalier Est- Hôpital Louis Pradel Bron cedex France 69677
11 Groupement Hospitalier Sud - Hôpital Bicêtre Le Kremlin Bicêtre cedex France 94275
12 Hopital Jeanne de Flandre - CHU Lille Lille Cedex France 59037
13 Hôpital de la Timone Marseille Cedex 05 France 13385
14 CHU de Nantes Site Hotel Dieu Nantes Cedex 1 France 44093
15 Vivantes Klinikum im Friedrichshain Berlin Germany 10249
16 Klinikum der Johann Wolfgang Goethe-Universitaet Frankfurt Germany 60590
17 Presidio Ospedaliero di Castelfranco Veneto Castelfranco Veneto Italy 31033
18 Azienda Ospedaliera Universitaria Careggi Firenze Italy 50134
19 Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico Milano Italy 20122
20 Ospedale San Bortolo di Vicenza Vicenza Italy 36100
21 Hospital Universitario La Paz Madrid Spain 28046
22 Hospital Regional Universitario de Malaga Malaga Spain 29010
23 Hospital Universitario de Salamanca Salamanca Spain 37007

Sponsors and Collaborators

  • Baxalta now part of Shire

Investigators

  • Study Director: Study Director, Shire

Study Documents (Full-Text)

None provided.

More Information

Additional Information:

Publications

None provided.
Responsible Party:
Baxalta now part of Shire
ClinicalTrials.gov Identifier:
NCT03185897
Other Study ID Numbers:
  • 201601
First Posted:
Jun 14, 2017
Last Update Posted:
Oct 25, 2021
Last Verified:
Oct 1, 2021
Individual Participant Data (IPD) Sharing Statement:
Yes
Plan to Share IPD:
Yes
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No
Additional relevant MeSH terms:
Hemophilia A
Hemophilia B

Study Results

No Results Posted as of Oct 25, 2021