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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.

Register:	ClinicalTrials.gov
Last refreshed on:	7 June 2021
Main ID:	NCT02502409
Date of registration:	13/07/2015
Prospective Registration:	No
Primary sponsor:	Skane University Hospital
Public title:	Natural History Study of Factor IX Treatment and Complications B-Natural
Scientific title:	Natural History Study of Factor IX Treatment and Complications
Date of first enrolment:	July 2015
Target sample size:	550
Recruitment status:	Active, not recruiting
URL:	https://clinicaltrials.gov/show/NCT02502409
Study type:	Observational
Study design:
Phase:

Countries of recruitment
Sweden	United States

Contacts

Name:	Jan Astermark, MD, PhD
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Affiliation:	Skåne University Hospital, Malmö

Name:	Manuela Carvalho, MD
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Affiliation:	Centro Hospitalar de São João, Porto, Portugal

Name:	Ulrike Reiss, MD
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Affiliation:	St. Jude Children's Research Hospital, Memphis, TN US

Name:	Cristina Tarango, MD
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Affiliation:	Cincinnati Children's Hospital Medical Center, Cincinnati, OH US

Name:	Raina Liesner, MD
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Affiliation:	Great Ormond Street Hospital for Children, London, UK

Name:	Munira Borhany, MD
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Affiliation:	National Institute of Blood Disease and Bone Marrow Transplantation, Karachi, Pakistan

Name:	Erik Berntorp, MD, PhD
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Affiliation:	Skåne University Hospital, Malmö

Name:	Stacy Croteau, MD
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Affiliation:	Boston Hemophilia Center, Boston, MA

Name:	Suchitra Acharya, MD
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Affiliation:	Cohen Children's Medical Center, New Hyde Park, NY

Name:	Eva Funding, MD
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Affiliation:	National University Hospital Copenhagen, Copenhagen, Denmark

Name:	Christine Knoll, MD
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Affiliation:	Phoenix Children's Hospital, Phoenix, AZ

Name:	Margaret Ragni, MD
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Affiliation:	University of Pittsburgh and Hemophilia Center of Western Pennsylvania, Pittburgh, PA US

Name:	Catherine McGuinn, MD
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Affiliation:	Weill Cornell Medical College, New York, NY

Name:	Susan Kearney, MD
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Affiliation:	Children's Hospitals and Clinics of Minnesota, Minneapolis, MN

Name:	Christine Kempton, MD
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Affiliation:	Emory University, Atlanta, Georgia

Name:	Amy D Shapiro, MD
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Affiliation:	Indiana Hemophilia &Thrombosis Center, Inc.

Name:	Roshni Kulkarni, MD
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Affiliation:	Michigan State University, East Lansing, MI

Name:	Michelle Witkop, DNP, FNP-BC
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Affiliation:	Munson Medical Center, Traverse City, MI, US

Name:	Johannes Oldenburg, MD, Ph.D
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Affiliation:	University Clinic, Bonn

Name:	Christoph Bidlingmaier, MD
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Affiliation:	Dr. v Hauner Children's University Hospital, Munich, Germany

Name:	Katharina Holstein, MD
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Affiliation:	University Medical Centre Hamburg-Eppendorf, Hamburg, Germany

Name:	Yasmina Abajas, MD
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Affiliation:	University of North Carolina Hemophilia Treatment Center, Chapel Hill, NC

Name:	Michael D Tarantino, MD
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Affiliation:	Bleeding and Clotting Disorders Institute, Peoria, IL US

Name:	Philip Kuriakose, MD
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Affiliation:	Henry Ford Health System, Detroit, MI

Name:	Alice J. Cohen, MD
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Affiliation:	Newark Beth Israel Medical Center, Newark, NJ

Key inclusion & exclusion 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

Age minimum: N/A
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Factor IX Deficiency

Intervention(s)

Other: Standard care with blood and urine sample collection

Primary Outcome(s)

Hemophilia treatment adherence [Time Frame: Baseline]

Joint assessment [Time Frame: Baseline]

Renal disorders [Time Frame: 6 months]

Inhibitory antibodies [Time Frame: Baseline]

Annualized bleeding rate [Time Frame: 6 months]

Health related quality of life [Time Frame: Baseline]

Non-inhibitory antibodies [Time Frame: Baseline]

Secondary Outcome(s)

Number of hospitalizations [Time Frame: 6 months]

Number of surgical procedures [Time Frame: 6 months]

Factor IX usage [Time Frame: 6 months]

number of days missed from school or work [Time Frame: 6 months]

Secondary ID(s)

B-Natural

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Indiana Hemophilia &Thrombosis Center, Inc.

Bioverativ Therapeutics Inc.

Swedish Orphan Biovitrum

Ethics review

Results

Results available:
Date Posted:
Date Completed:
URL:

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