Natural History Study of Factor IX Treatment and Complications

NCT ID: NCT02502409

Last Updated: 2021-05-25

Basic Information

• Recruitment Status: UNKNOWN
• Total Enrollment: 550 participants
• Study Classification: OBSERVATIONAL
• Study Start Date: 2015-07-31
• Study Completion Date: 2021-12-31

Brief Summary

This study will examine two groups of subjects with factor IX (FIX) deficiency: 1) those with a current or history of inhibitors to FIX, and; 2) groups of two or more affected brothers, with or without inhibitors. The overall goal is to characterize the study groups in terms of their medical history, their patterns of bleeding, their care, quality of life, and complications including the development of joint disease, inhibitory antibodies to FIX, use of immune tolerance induction (ITI) and outcome.

Detailed Description

Hemophilia B, FIX deficiency, is the second most common type of hemophilia, occurring in about one in 25,000 male births. This disease is in some ways more complex than hemophilia A, and is less well understood. Differences include a lower incidence and a greater risk of side effects to treatment, for example, allergic reactions and kidney disease. This study will examine two groups of subjects with FIX deficiency - those with a current or history of inhibitors to FIX, and groups of two or more affected brothers, with or without inhibitors. The overall goal is to characterize the study group in terms of their medical history, their patterns of bleeding, their care, quality of life, and complications including the development of inhibitory antibodies to FIX, allergies, kidney, and joint disease.

Conditions

Factor IX Deficiency

Study Design

• Observational Model Type: OTHER
• Study Time Perspective: PROSPECTIVE

Interventions

Standard care with blood and urine sample collection

Intervention Type: OTHER

Eligibility Criteria

Inclusion Criteria

1. A consent approved by the appropriate Institutional Review Board (IRB)/Independent Ethics Committee (IEC) has been obtained from the subject or his legally acceptable representative

2. Subject has FIX deficiency AND

• Is part of an affected brother pair/group that will also enroll; AND/OR
• Has a current or history of inhibitor, defined as >0.6 Bethesda units (BU)

Exclusion Criteria

1. Subject has another congenital bleeding disorder

2. Subject is a carrier of hemophilia B with factor level >0.40 IU/mL

• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Indiana Hemophilia &Thrombosis Center, Inc.

OTHER

Sponsor Role: collaborator

Bioverativ Therapeutics Inc.

INDUSTRY

Sponsor Role: collaborator

Swedish Orphan Biovitrum

INDUSTRY

Sponsor Role: collaborator

Skane University Hospital

OTHER

Sponsor Role: lead

Responsible Party

Sharyne M. Donfield, Ph.D.

Principal Investigator, Data Coordinating Center, Rho, Inc.

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Erik Berntorp, MD, PhD

Role: STUDY_DIRECTOR

Skåne University Hospital, Malmö

Amy D Shapiro, MD

Role: STUDY_DIRECTOR

Indiana Hemophilia &Thrombosis Center, Inc.

Jan Astermark, MD, PhD

Role: STUDY_DIRECTOR

Skåne University Hospital, Malmö

Christine Knoll, MD

Role: PRINCIPAL_INVESTIGATOR

Phoenix Children's Hospital, Phoenix, AZ

Yasmina Abajas, MD

Role: PRINCIPAL_INVESTIGATOR

University of North Carolina Hemophilia Treatment Center, Chapel Hill, NC

Catherine McGuinn, MD

Role: PRINCIPAL_INVESTIGATOR

Weill Cornell Medical College, New York, NY

Munira Borhany, MD

Role: PRINCIPAL_INVESTIGATOR

National Institute of Blood Disease and Bone Marrow Transplantation, Karachi, Pakistan

Philip Kuriakose, MD

Role: PRINCIPAL_INVESTIGATOR

Henry Ford Health System, Detroit, MI

Eva Funding, MD

Role: PRINCIPAL_INVESTIGATOR

National University Hospital Copenhagen, Copenhagen, Denmark

Stacy Croteau, MD

Role: PRINCIPAL_INVESTIGATOR

Boston Hemophilia Center, Boston, MA

Christine Kempton, MD

Role: PRINCIPAL_INVESTIGATOR

Emory University, Atlanta, Georgia

Susan Kearney, MD

Role: PRINCIPAL_INVESTIGATOR

Children's Hospitals and Clinics of Minnesota, Minneapolis, MN

Suchitra Acharya, MD

Role: PRINCIPAL_INVESTIGATOR

Cohen Children's Medical Center, New Hyde Park, NY

Roshni Kulkarni, MD

Role: PRINCIPAL_INVESTIGATOR

Michigan State University, East Lansing, MI

Raina Liesner, MD

Role: PRINCIPAL_INVESTIGATOR

Great Ormond Street Hospital for Children, London, UK

Christoph Bidlingmaier, MD

Role: PRINCIPAL_INVESTIGATOR

Dr. v Hauner Children's University Hospital, Munich, Germany

Alice J. Cohen, MD

Role: PRINCIPAL_INVESTIGATOR

Newark Beth Israel Medical Center, Newark, NJ

Manuela Carvalho, MD

Role: PRINCIPAL_INVESTIGATOR

Centro Hospitalar de São João, Porto, Portugal

Margaret Ragni, MD

Role: PRINCIPAL_INVESTIGATOR

University of Pittsburgh and Hemophilia Center of Western Pennsylvania, Pittburgh, PA US

Ulrike Reiss, MD

Role: PRINCIPAL_INVESTIGATOR

St. Jude Children's Research Hospital, Memphis, TN US

Michelle Witkop, DNP, FNP-BC

Role: PRINCIPAL_INVESTIGATOR

Munson Medical Center, Traverse City, MI, US

Katharina Holstein, MD

Role: PRINCIPAL_INVESTIGATOR

University Medical Centre Hamburg-Eppendorf, Hamburg, Germany

Cristina Tarango, MD

Role: PRINCIPAL_INVESTIGATOR

Cincinnati Children's Hospital Medical Center, Cincinnati, OH US

Michael D Tarantino, MD

Role: PRINCIPAL_INVESTIGATOR

Bleeding and Clotting Disorders Institute, Peoria, IL US

Johannes Oldenburg, MD, Ph.D

Role: PRINCIPAL_INVESTIGATOR

University Clinic, Bonn

Locations

Indiana Hemophilia & Thrombosis Center

Indianapolis, Indiana, United States

Countries

United States

References

Shapiro AD, Ragni MV, Borhany M, Abajas YL, Tarantino MD, Holstein K, Croteau SE, Liesner R, Tarango C, Carvalho M, McGuinn C, Funding E, Kempton CL, Bidlingmaier C, Cohen A, Oldenburg J, Kearney S, Knoll C, Kuriakose P, Acharya S, Reiss UM, Kulkarni R, Witkop M, Lethagen S, Donfield S, LeBeau P, Berntorp E, Astermark J. Natural history study of factor IX deficiency with focus on treatment and complications (B-Natural). Haemophilia. 2021 Jan;27(1):49-59. doi: 10.1111/hae.14139. Epub 2020 Dec 5.

Reference Type: BACKGROUND

PMID: 33278853 (View on PubMed)

Other Identifiers

B-Natural

Identifier Type: -

Identifier Source: org_study_id