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Natural History Study of Factor IX Treatment and Complications (B-Natural)

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ClinicalTrials.gov Identifier: NCT02502409
Recruitment Status : Active, not recruiting
First Posted : July 20, 2015
Last Update Posted : April 17, 2018
Sponsor:
Collaborators:
Indiana Hemophilia &Thrombosis Center, Inc.
University of Bonn
Biogen
Swedish Orphan Biovitrum
Information provided by (Responsible Party):
Sharyne M. Donfield, Ph.D., Skane University Hospital

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.

Condition or disease Intervention/treatment
Factor IX Deficiency Other: Standard care with blood and urine sample collection

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.

Study Type : Observational
Estimated Enrollment : 550 participants
Observational Model: Other
Time Perspective: Prospective
Official Title: Natural History Study of Factor IX Treatment and Complications
Study Start Date : July 2015
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : May 2021

Resource links provided by the National Library of Medicine





Primary Outcome Measures :
  1. Inhibitory antibodies [ Time Frame: Baseline ]
    Current or history of inhibitors

  2. Annualized bleeding rate [ Time Frame: 6 months ]
    Overall and by bleeding site

  3. Joint assessment [ Time Frame: Baseline ]
    Range of motion

  4. Renal disorders [ Time Frame: 6 months ]
  5. Hemophilia treatment adherence [ Time Frame: Baseline ]
    Validated Hemophilia Regimen Treatment Adherence Scale--Prophylaxis (VERITAS-Pro), Validated Hemophilia Regimen Treatment Adherence Scale - PRN (VERITAS-PRN)

  6. Health related quality of life [ Time Frame: Baseline ]
    European Quality of Life - 5 Dimensions (EQ5D)

  7. Non-inhibitory antibodies [ Time Frame: Baseline ]

Secondary Outcome Measures :
  1. Factor IX usage [ Time Frame: 6 months ]
  2. Number of hospitalizations [ Time Frame: 6 months ]
  3. Number of surgical procedures [ Time Frame: 6 months ]
  4. number of days missed from school or work [ Time Frame: 6 months ]

Biospecimen Retention:   Samples With DNA
Serum, plasma, cells


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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
The population includes groups of two or more affected brothers, with or without a history of inhibitors, who share(d) one or both biological parents; and individuals with a history of an inhibitor. Most affected brother pairs will be concordant for no inhibitor and will serve as a control group for those with inhibitors. The study is open to subjects with mild (0.05-0.40 IU/mL), moderate (0.01-<0.05 IU/mL), or severe (<0.01 IU/mL) FIX deficiency. Females meeting the eligibility criteria may participate. There are no lower or upper age limits. Type of treatment, regimen, dosing and product(s) used are at the discretion of the investigator.
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02502409


Locations
United States, North Carolina
Rho, Inc.
Chapel Hill, North Carolina, United States, 27517
Sponsors and Collaborators
Skane University Hospital
Indiana Hemophilia &Thrombosis Center, Inc.
University of Bonn
Biogen
Swedish Orphan Biovitrum
Investigators
Study Director: Erik Berntorp, MD, PhD Skåne University Hospital, Malmö
Study Director: Amy D Shapiro, MD Indiana Hemophilia &Thrombosis Center, Inc.
Study Director: Jan Astermark, MD, PhD Skåne University Hospital, Malmö
Principal Investigator: Christine Knoll, MD Phoenix Children's Hospital, Phoenix, AZ
Principal Investigator: Yasmina Abajas, MD University of North Carolina Hemophilia Treatment Center, Chapel Hill, NC
Principal Investigator: Catherine McGuinn, MD Weill Cornell Medical College, New York, NY
Principal Investigator: Munira Borhany, MD National Institute of Blood Disease and Bone Marrow Transplantation, Karachi, Pakistan
Principal Investigator: Philip Kuriakose, MD Henry Ford Health System, Detroit, MI
Principal Investigator: Eva Funding, MD National University Hospital Copenhagen, Copenhagen, Denmark
Principal Investigator: Stacy Croteau, MD Boston Hemophilia Center, Boston, MA
Principal Investigator: Christine Kempton, MD Emory University, Atlanta, Georgia
Principal Investigator: Susan Kearney, MD Children's Hospitals and Clinics of Minnesota, Minneapolis, MN
Principal Investigator: Suchitra Acharya, MD Cohen Children's Medical Center, New Hyde Park, NY
Principal Investigator: Roshni Kulkarni, MD Michigan State University, East Lansing, MI
Principal Investigator: Raina Liesner, MD Great Ormond Street Hospital for Children, London, UK
Principal Investigator: Christoph Bidlingmaier, MD Dr. v Hauner Children's University Hospital, Munich, Germany
Principal Investigator: Alice J. Cohen, MD Newark Beth Israel Medical Center, Newark, NJ
Principal Investigator: Manuela Carvalho, MD Centro Hospitalar de São João, Porto, Portugal
Principal Investigator: Margaret Ragni, MD University of Pittsburgh and Hemophilia Center of Western Pennsylvania, Pittburgh, PA US
Principal Investigator: Ulrike Reiss, MD St. Jude Children's Research Hospital, Memphis, TN US
Principal Investigator: Michelle Witkop, DNP, FNP-BC Munson Medical Center, Traverse City, MI, US
Principal Investigator: Katharina Holstein, MD University Medical Centre Hamburg-Eppendorf, Hamburg, Germany
Principal Investigator: Cristina Tarango, MD Cincinnati Children's Hospital Medical Center, Cincinnati, OH US
Principal Investigator: Michael D Tarantino, MD Bleeding and Clotting Disorders Institute, Peoria, IL US
Principal Investigator: Johannes Oldenburg, MD, Ph.D University Clinic, Bonn

Responsible Party: Sharyne M. Donfield, Ph.D., Principal Investigator, Data Coordinating Center, Rho, Inc., Skane University Hospital
ClinicalTrials.gov Identifier: NCT02502409     History of Changes
Other Study ID Numbers: B-Natural
First Posted: July 20, 2015    Key Record Dates
Last Update Posted: April 17, 2018
Last Verified: April 2018

Keywords provided by Sharyne M. Donfield, Ph.D., Skane University Hospital:
Hemophilia B
Hemophilia B with Inhibitors

Additional relevant MeSH terms:
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked