Factor IX DeficiencyB-NaturalNCT02502409
Natural History Study of Factor IX Treatment and Complications
An observational study of Factor IX Deficiency, run by Skane University Hospital at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Factor IX Deficiency
- How many people
- 550 participants
- Where
- 1 siteUnited States
- Study dates
- Jul 2015to Dec 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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
- 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)
Who cannot join
- Subject has another congenital bleeding disorder
- Subject is a carrier of hemophilia B with factor level >0.40 IU/mL
Trial locations and contacts
1 site in 1 country.
United States1 site
Indiana Hemophilia & Thrombosis Center
Indianapolis, Indiana, 46260
About this study
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.
Read the 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
- Being tested
- Standard care with blood and urine sample collection
- Keywords
- Hemophilia B, Hemophilia B with Inhibitors
- Medical subject headings
- Hemophilia B
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Standard care with blood and urine sample collectionOther
What is being measured
The primary outcome is the main question the study is designed to answer.
Inhibitory antibodies
Current or history of inhibitors
Measured: Baseline
Annualized bleeding rate
Overall and by bleeding site
Measured: 6 months
Joint assessment
Range of motion
Measured: Baseline
Renal disorders
Reported subject and family history of renal disease
Measured: 6 months
Hemophilia treatment adherence
Validated Hemophilia Regimen Treatment Adherence Scale--Prophylaxis (VERITAS-Pro), Validated Hemophilia Regimen Treatment Adherence Scale - PRN (VERITAS-PRN)
Measured: Baseline
Health related quality of life
European Quality of Life - 5 Dimensions (EQ5D)
Measured: Baseline
Non-inhibitory antibodies
Measured at central laboratory
Measured: Baseline
Secondary outcomes (4)
Factor IX usage
Treatment history with FIX replacement
Measured: 6 months
Number of hospitalizations
Hospitalizations reported during 6 month follow up period
Measured: 6 months
Number of surgical procedures
Surgical procedures reported during 6 month follow up period
Measured: 6 months
number of days missed from school or work
Days missed from school or work during 6 month follow up period
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Jul 2015 (estimated)
- Study startJul 2015
- Primary completionJun 2019
- Study completionDec 2021 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Factor IX DeficiencyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Prospective
- Enrolment
- 550 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Skane University Hospital
- Sponsor type
- University, hospital or other
- Collaborators
- Indiana Hemophilia &Thrombosis Center, Inc., Bioverativ Therapeutics Inc., Swedish Orphan Biovitrum
- Responsible party
- Principal investigator, Sharyne M. Donfield, Ph.D., Skane University Hospital
- Investigators
- Erik Berntorp, MD, PhD, Study director, Skåne University Hospital, MalmöAmy D Shapiro, MD, Study director, Indiana Hemophilia &Thrombosis Center, Inc.Jan Astermark, MD, PhD, Study director, Skåne University Hospital, MalmöChristine Knoll, MD, Principal investigator, Phoenix Children's Hospital, Phoenix, AZYasmina Abajas, MD, Principal investigator, University of North Carolina Hemophilia Treatment Center, Chapel Hill, NCCatherine McGuinn, MD, Principal investigator, Weill Cornell Medical College, New York, NYMunira Borhany, MD, Principal investigator, National Institute of Blood Disease and Bone Marrow Transplantation, Karachi, PakistanPhilip Kuriakose, MD, Principal investigator, Henry Ford Health System, Detroit, MI
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02502409
- Sponsor's protocol ID
- B-Natural
- Acronym
- B-Natural
- Status last verified
- May 2021
- First submitted
- 13 Jul 2015
- Last updated
- 25 May 2021
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 33278853
Study at a glance
From the registry record, last updated by the sponsor on 25 May 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Natural History Study of Factor IX Treatment and Complications
- ClinicalTrials.gov ID
- NCT02502409
- Acronym / sponsor ID
- B-Natural / B-Natural
- Conditions
- Factor IX Deficiency
- Keywords
- Hemophilia B, Hemophilia B with Inhibitors
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 550 participants (estimated)
- Lead sponsor
- Skane University HospitalUniversity, hospital or other
- Collaborators
- Indiana Hemophilia &Thrombosis Center, Inc., Bioverativ Therapeutics Inc., Swedish Orphan Biovitrum
- Study start
- Jul 2015
- Primary completion
- Jun 2019 (actual)
- Study completion
- Dec 2021 (estimated)
- First posted
- 20 Jul 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Other
- Time perspective
- Prospective
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (4 listed)
- 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
- Subject has FIX deficiency AND
Key exclusion criteria (2 listed)
- Subject has another congenital bleeding disorder
- Subject is a carrier of hemophilia B with factor level >0.40 IU/mL
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Standard care with blood and urine sample collection | Other | – |
Primary outcome
Inhibitory antibodies
Time frame: Baseline
Current or history of inhibitors
Annualized bleeding rate
Time frame: 6 months
Overall and by bleeding site
Joint assessment
Time frame: Baseline
Range of motion
Renal disorders
Time frame: 6 months
Reported subject and family history of renal disease
Hemophilia treatment adherence
Time frame: Baseline
Validated Hemophilia Regimen Treatment Adherence Scale--Prophylaxis (VERITAS-Pro), Validated Hemophilia Regimen Treatment Adherence Scale - PRN (VERITAS-PRN)
Health related quality of life
Time frame: Baseline
European Quality of Life - 5 Dimensions (EQ5D)
Non-inhibitory antibodies
Time frame: Baseline
Measured at central laboratory
4 secondary outcomes
Factor IX usage
Time frame: 6 months
Number of hospitalizations
Time frame: 6 months
Number of surgical procedures
Time frame: 6 months
number of days missed from school or work
Time frame: 6 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 25 May 2021.
- Background: PubMed 33278853Shapiro 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.
Related trials for Factor IX Deficiency
Not yet recruiting
Safety of Intraosseous Anaesthesia in Children With Haemophilia
Haemophilia A, Haemophilia B
RecruitingPhase 3
Efficacy and Safety of 4F-PCC (4-Factor Prothrombin Complex Concentrate) in Adult Patients Undergoing Complex Cardiovascular Surgery With Cardiopulmonary Bypass (CPB)
Complex Cardiovascular Surgery With Cardiopulmonary Bypass
Enrolling by invitationObservational study
Adverse Event Data Collection From External Registries on Nonacog Beta Pegol
Haemophilia B
Active, not recruitingPhase 3
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia Without Inhibitors
Haemophilia A Without Inhibitors, Haemophilia B Without Inhibitors
Active, not recruitingPhase 3
Research Study to Look at How Well the Drug Concizumab Works in Your Body if You Have Haemophilia With Inhibitors
Haemophilia A With Inhibitors, Haemophilia B With Inhibitors
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Erik Berntorp, MD, PhD study director
Skåne University Hospital, Malmö
Amy D Shapiro, MD study director
Indiana Hemophilia &Thrombosis Center, Inc.
Jan Astermark, MD, PhD study director
Skåne University Hospital, Malmö
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Factor IX Deficiency, run by Skane University Hospital at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 25 May 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Inhibitory antibodies (time frame: Baseline). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02502409, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 25 May 2021.