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

May 20, 2021 updated by: Sharyne M. Donfield, Ph.D., Skane University Hospital
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.

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Active, not recruiting

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

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

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Anticipated)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
550

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United States
    • Indiana
      • Indianapolis, Indiana, United States, 46260
        • Indiana Hemophilia & Thrombosis center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Other
  • Time Perspectives: Prospective

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Inhibitory antibodies
Time Frame: Baseline
Current or history of inhibitors
Baseline
Annualized bleeding rate
Time Frame: 6 months
Overall and by bleeding site
6 months
Joint assessment
Time Frame: Baseline
Range of motion
Baseline
Renal disorders
Time Frame: 6 months
Reported subject and family history of renal disease
6 months
Hemophilia treatment adherence
Time Frame: Baseline
Validated Hemophilia Regimen Treatment Adherence Scale--Prophylaxis (VERITAS-Pro), Validated Hemophilia Regimen Treatment Adherence Scale - PRN (VERITAS-PRN)
Baseline
Health related quality of life
Time Frame: Baseline
European Quality of Life - 5 Dimensions (EQ5D)
Baseline
Non-inhibitory antibodies
Time Frame: Baseline
Measured at central laboratory
Baseline

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Factor IX usage
Time Frame: 6 months
Treatment history with FIX replacement
6 months
Number of hospitalizations
Time Frame: 6 months
Hospitalizations reported during 6 month follow up period
6 months
Number of surgical procedures
Time Frame: 6 months
Surgical procedures reported during 6 month follow up period
6 months
number of days missed from school or work
Time Frame: 6 months
Days missed from school or work during 6 month follow up period
6 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
Skane University Hospital

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
Indiana Hemophilia &Thrombosis Center, Inc.
Bioverativ Therapeutics Inc.
Swedish Orphan Biovitrum

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • 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

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
July 1, 2015

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
June 1, 2019

Study Completion (Anticipated)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
December 1, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
July 13, 2015

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 16, 2015

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
July 20, 2015

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
May 25, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 20, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
May 1, 2021

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • B-Natural

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