Natural History Study of Factor IX Treatment and Complications

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.

Conditions

• Factor IX Deficiency

Interventions

OTHER

Standard care with blood and urine sample collection

Sponsors & Collaborators

• Indiana Hemophilia &Thrombosis Center, Inc.

  collaborator OTHER

• Bioverativ Therapeutics Inc.

  collaborator INDUSTRY

• Swedish Orphan Biovitrum

  collaborator INDUSTRY

• Skane University Hospital

  lead OTHER

Principal Investigators

• Erik Berntorp, MD, PhD · Skåne University Hospital, Malmö

• Amy D Shapiro, MD · Indiana Hemophilia &Thrombosis Center, Inc.

• Jan Astermark, MD, PhD · Skåne University Hospital, Malmö

• Christine Knoll, MD · Phoenix Children's Hospital, Phoenix, AZ

• Yasmina Abajas, MD · University of North Carolina Hemophilia Treatment Center, Chapel Hill, NC

• Catherine McGuinn, MD · Weill Cornell Medical College, New York, NY

• Munira Borhany, MD · National Institute of Blood Disease and Bone Marrow Transplantation, Karachi, Pakistan

• Philip Kuriakose, MD · Henry Ford Health System, Detroit, MI

• Eva Funding, MD · National University Hospital Copenhagen, Copenhagen, Denmark

• Stacy Croteau, MD · Boston Hemophilia Center, Boston, MA

• Christine Kempton, MD · Emory University, Atlanta, Georgia

• Susan Kearney, MD · Children's Hospitals and Clinics of Minnesota, Minneapolis, MN

• Suchitra Acharya, MD · Cohen Children's Medical Center, New Hyde Park, NY

• Roshni Kulkarni, MD · Michigan State University, East Lansing, MI

• Raina Liesner, MD · Great Ormond Street Hospital for Children, London, UK

• Christoph Bidlingmaier, MD · Dr. v Hauner Children's University Hospital, Munich, Germany

• Alice J. Cohen, MD · Newark Beth Israel Medical Center, Newark, NJ

• Manuela Carvalho, MD · Centro Hospitalar de São João, Porto, Portugal

• Margaret Ragni, MD · University of Pittsburgh and Hemophilia Center of Western Pennsylvania, Pittburgh, PA US

• Ulrike Reiss, MD · St. Jude Children's Research Hospital, Memphis, TN US

• Michelle Witkop, DNP, FNP-BC · Munson Medical Center, Traverse City, MI, US

• Katharina Holstein, MD · University Medical Centre Hamburg-Eppendorf, Hamburg, Germany

• Cristina Tarango, MD · Cincinnati Children's Hospital Medical Center, Cincinnati, OH US

• Michael D Tarantino, MD · Bleeding and Clotting Disorders Institute, Peoria, IL US

• Johannes Oldenburg, MD, Ph.D · University Clinic, Bonn

Eligibility

Sex

ALL

Healthy Volunteers

No

Timeline & Regulatory

Start

2015-07-31

Primary Completion

2019-06-30

Completion

2021-12-31

Countries

• United States

Study Locations