B-Natural
Research type
Research Study
Full title
Natural History Study of Factor IX Treatment and Complications (B-Natural)
IRAS ID
202331
Contact name
Sharyne Donfield
Contact email
Sponsor organisation
Rho, Inc.
Clinicaltrials.gov Identifier
Duration of Study in the UK
2 years, 0 months, 0 days
Research summary
Hemophilia B, factor IX (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: 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.\n\nThis is an international multi-center, prospective, observational study. Type of treatment, including regimen, dosing, and type of product(s) used will be at the discretion of the investigator. The study is open to subjects of all ages with mild (0.05-0.40 IU/mL), moderate (0.01-<0.05 IU/mL), or severe (<0.01 IU/mL) clotting factor deficiency. \n\nAfter enrollment, participants will be followed for six months. A log of bleeding episodes and frequency and dosage of infusions will be maintained throughout the study period. \n\nA hemophilia-related history, a history of other chronic diseases, and use of medications, will be taken. Blood pressure will be measured. Joint assessment, quality of life assessment, and adherence to hemophilia treatment will be measured. Days missed from school or work and hospitalizations due to hemophilia will be recorded.\n\nA urine sample and blood samples will be collected at entry for assessment of kidney function, measurement of non-inhibitory antibodies, F9 gene mutation (if not already determined), and HLA typing. \n\n
REC name
London - Bloomsbury Research Ethics Committee
REC reference
16/LO/1268
Date of REC Opinion
1 Nov 2016
REC opinion
Further Information Favourable Opinion