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

Hemophilia A Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03185897
• Other study ID #: 201601
• Status: Completed
• Start date: June 14, 2017
• Est. completion date: March 17, 2021

Study information

• Verified date: October 2021
• Source: Takeda
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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%).

Recruitment information / eligibility

• Status: Completed
• Enrollment: 242
• Est. completion date: March 17, 2021
• Est. primary completion date: March 17, 2021
• Accepts healthy volunteers: No
• Gender: Male
• Age group: 18 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Hemophilia A
• Hemophilia B

Intervention

Other:
• Non-treatment, seroprevalence
Non-treatment study examining the prevalence of preexisting immunity to adeno-associated virus (AAV)

Locations

Country	Name	City	State
Austria	AKH - Medizinische Universität Wien	Vienna
France	Hôpital Morvan	Brest Cedex
France	Groupement Hospitalier Est- Hôpital Louis Pradel	Bron cedex
France	Groupement Hospitalier Sud - Hôpital Bicêtre	Le Kremlin Bicêtre cedex
France	Hopital Jeanne de Flandre - CHU Lille	Lille Cedex
France	Hôpital de la Timone	Marseille Cedex 05
France	CHU de Nantes Site Hotel Dieu	Nantes Cedex 1
Germany	Vivantes Klinikum im Friedrichshain	Berlin
Germany	Klinikum der Johann Wolfgang Goethe-Universitaet	Frankfurt
Italy	Presidio Ospedaliero di Castelfranco Veneto	Castelfranco Veneto
Italy	Azienda Ospedaliera Universitaria Careggi	Firenze
Italy	Fondazione IRCCS CA' Granda Ospedale Maggiore Policlinico	Milano
Italy	Ospedale San Bortolo di Vicenza	Vicenza
Spain	Hospital Universitario La Paz	Madrid
Spain	Hospital Regional Universitario de Malaga	Malaga
Spain	Hospital Universitario de Salamanca	Salamanca
United States	University of Colorado Hemophilia & Thrombosis Center	Aurora	Colorado
United States	Medical University of South Carolina (MUSC)	Charleston	South Carolina
United States	Cincinnati Children's Hospital Medical Center	Cincinnati	Ohio
United States	Michigan State University	East Lansing	Michigan
United States	Gulf States Hemophilia and Thrombophilia Center	Houston	Texas
United States	Orthopaedic Hemophilia Treatment Center	Los Angeles	California
United States	University of Washington	Seattle	Washington

Sponsors (1)

Lead Sponsor	Collaborator
Baxalta now part of Shire

Countries where clinical trial is conducted

United States,  Austria,  France,  Germany,  Italy,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Prevalence of neutralizing antibodies (NAb) - Baseline visit	Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes	Baseline visit
Primary	Prevalence of neutralizing antibodies (NAb) - Year 1 Visit	Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes	Year 1 visit
Primary	Prevalence of neutralizing antibodies (NAb) - Year 2 Visit	Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes	Year 2 visit
Primary	Prevalence of neutralizing antibodies (NAb) - Year 3 Visit	Prevalence of NAb to naturally occurring adeno-associated virus (AAV) serotypes	Year 3 visit
Secondary	Prevalence of NAb to AAV including AAV2 and AAV8	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
Secondary	Prevalence of binding antibodies to AAV, including AAV8 and AAV2	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
Secondary	T-cell mediated immune response to AAV8	T-cell mediated immune response to AAV8	Baseline visit, Year 1 visit, Year 2 visit, and Year 3 visit

External Links

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