Natural History Study of Factor IX Treatment and Complications

Factor IX Deficiency Clinical Trial

— B-Natural  

Official title:

Natural History Study of Factor IX Treatment and Complications

Clinical Trial Details — Status: Active, not recruiting

Administrative data

• NCT number: NCT02502409
• Other study ID #: B-Natural
• Status: Active, not recruiting
• Start date: July 2015
• Est. completion date: December 2021

Study information

• Verified date: May 2021
• Source: Skane University Hospital
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical 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. 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.

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.

Recruitment information / eligibility

• Status: Active, not recruiting
• Enrollment: 550
• Est. completion date: December 2021
• Est. primary completion date: June 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A and older

Eligibility

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

Related Conditions & MeSH terms

• Factor IX Deficiency
• Hemophilia B

Intervention

Other:
• Standard care with blood and urine sample collection

Locations

Country	Name	City	State
United States	Indiana Hemophilia & Thrombosis Center	Indianapolis	Indiana

Sponsors (4)

Lead Sponsor	Collaborator
Skane University Hospital	Bioverativ Therapeutics Inc., Indiana Hemophilia &Thrombosis Center, Inc., Swedish Orphan Biovitrum

Country where clinical trial is conducted

United States, 

References & Publications (1)

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. — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Inhibitory antibodies	Current or history of inhibitors	Baseline
Primary	Annualized bleeding rate	Overall and by bleeding site	6 months
Primary	Joint assessment	Range of motion	Baseline
Primary	Renal disorders	Reported subject and family history of renal disease	6 months
Primary	Hemophilia treatment adherence	Validated Hemophilia Regimen Treatment Adherence Scale--Prophylaxis (VERITAS-Pro), Validated Hemophilia Regimen Treatment Adherence Scale - PRN (VERITAS-PRN)	Baseline
Primary	Health related quality of life	European Quality of Life - 5 Dimensions (EQ5D)	Baseline
Primary	Non-inhibitory antibodies	Measured at central laboratory	Baseline
Secondary	Factor IX usage	Treatment history with FIX replacement	6 months
Secondary	Number of hospitalizations	Hospitalizations reported during 6 month follow up period	6 months
Secondary	Number of surgical procedures	Surgical procedures reported during 6 month follow up period	6 months
Secondary	number of days missed from school or work	Days missed from school or work during 6 month follow up period	6 months