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

NCT03185897 | Completed

• 1 other identifier: 201601
• Study Type: observational
• Target: 242
• Locations: 6 countries
• Sites: 23

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%).

Conditions

Hemophilia A; Hemophilia B

Outcome Measures

Primary Outcomes (4)

• Prevalence of neutralizing antibodies (NAb) - Baseline visit

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

  Baseline visit

• Prevalence of neutralizing antibodies (NAb) - 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

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

  Year 2 visit

• Prevalence of neutralizing antibodies (NAb) - Year 3 Visit

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

  Year 3 visit

Secondary Outcomes (3)

• Prevalence of NAb to 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

  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

Study Arms (2)

Hemophilia A

Participants with hemophilia A

Other: Non-treatment, seroprevalence

Hemophilia B

Participants with hemophilia B

Other: Non-treatment, seroprevalence

Interventions

Non-treatment, seroprevalence OTHER

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

Hemophilia A; Hemophilia B

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: male
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

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

You may qualify if:

• 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.

You may not qualify if:

• 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.
• Has a known immune deficiency other than HIV.
• Has lymphocyte or plasma cell malignancies.
• Participant is a family member or employee of the investigator.

Sponsors & Collaborators

• Baxalta now part of Shire: lead

Study Sites (23)

Orthopaedic Hemophilia Treatment Center

Los Angeles, California, 90007, United States

Location

University of Colorado Hemophilia & Thrombosis Center

Aurora, Colorado, 80045, United States

Location

Michigan State University

East Lansing, Michigan, 48824, United States

Location

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Medical University of South Carolina (MUSC)

Charleston, South Carolina, 29425, United States

Location

Gulf States Hemophilia and Thrombophilia Center

Houston, Texas, 77030, United States

Location

University of Washington

Seattle, Washington, 98104, United States

Location

AKH - Medizinische Universität Wien

Vienna, 1090, Austria

Location

Hôpital Morvan

Brest, 29609, France

Location

Groupement Hospitalier Est- Hôpital Louis Pradel

Bron, 69677, France

Location

Groupement Hospitalier Sud - Hôpital Bicêtre

Le Kremlin-Bicêtre, 94275, France

Location

Hopital Jeanne de Flandre - CHU Lille

Lille, 59037, France

Location

Hôpital de la Timone

Marseille, 13385, France

Location

CHU de Nantes Site Hotel Dieu

Nantes, 44093, France

Location

Vivantes Klinikum im Friedrichshain

Berlin, 10249, Germany

Location

Klinikum der Johann Wolfgang Goethe-Universitaet

Frankfurt, 60590, Germany

Location

Presidio Ospedaliero di Castelfranco Veneto

Castelfranco Veneto, 31033, Italy

Location

Azienda Ospedaliera Universitaria Careggi

Florence, 50134, Italy

Location

Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico

Milan, 20122, Italy

Location

Ospedale San Bortolo di Vicenza

Vicenza, 36100, Italy

Location

Hospital Universitario La Paz

Madrid, 28046, Spain

Location

Hospital Regional Universitario de Malaga

Málaga, 29010, Spain

Location

Hospital Universitario de Salamanca

Salamanca, 37007, Spain

Location

Related Links

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

MeSH Terms

Conditions

Hemophilia A; Hemophilia B

Interventions

Seroepidemiologic Studies

Condition Hierarchy (Ancestors)

Blood Coagulation Disorders, Inherited; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Coagulation Protein Disorders; Hemorrhagic Disorders; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Genetic Diseases, X-Linked

Intervention Hierarchy (Ancestors)

Epidemiologic Studies; Epidemiologic Study Characteristics; Epidemiologic Methods; Investigative Techniques; Health Care Evaluation Mechanisms; Quality of Health Care; Health Care Quality, Access, and Evaluation; Public Health; Environment and Public Health

Study Officials

• Study Director

  Shire

  STUDY DIRECTOR

Study Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: May 31, 2017
• First Posted: June 14, 2017
• Study Start: June 14, 2017
• Primary Completion: March 17, 2021
• Study Completion: March 17, 2021
• Last Updated: October 25, 2021

Record last verified: 2021-10

Data Sharing

• IPD Sharing: Will share
• Shared Documents: STUDY PROTOCOL, SAP, ICF, CSR
• 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.

Locations

US (7); AU (1); DE (2); IT (4); FR (6); ES (3)