A Post authorization safety surveillance registry with BeneFIX in hemophilia B patients in usual care settings

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)
• Data collection methods:
  Primary data collection

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  This is a non-interventional, voluntary prospective registry study conducted in major hemophilia treatment centers in China.

Study drug and medical condition

• Name of medicine:
  BENEFIX
• Medical condition to be studied:
  Haemophilia B without inhibitors

Population studied

• Short description of the study population:
  Any patient with Hemophilia B in China who is eligible and willing to participate in the registry.; Patients must meet all of the following inclusion criteria to be eligible for inclusion in the study:; 1. Male subjects of all ages and severity with Hemophilia B.; 2. Subjects using or intending to use BeneFIX for Factor IX replacement therapy.; 3. Subjects/parents/legal representatives must be able to comply with registry procedures (informed consent/assent process, clinical visits, reporting of infusion and bleed data, reporting of adverse events, etc).; 4. Evidence of a personally signed and dated informed consent document indicating that the patient (or a legally acceptable representative) has been informed of all pertinent aspects of the study.
• Age groups:
  Term newborn infants (0 – 27 days)

  Infants and toddlers (28 days – 23 months)

  Children (2 to < 12 years)

  Adolescents (12 to < 18 years)

  Adults (18 to < 46 years)

  Adults (46 to < 65 years)

  Adults (65 to < 75 years)

  Adults (75 to < 85 years)

  Adults (85 years and over)
• Special population of interest:
  Hepatic impaired

  Renal impaired
• Estimated number of subjects:
  60

Study design details

• Main study objective:
  Primary study objective is to evaluate the product medically important events (FIX inhibitor development, FIX hypersensitivity allergic reaction, thrombogenicity, lack of effect and red blood cell RBCagglutination ) in Chinese hemophilia B patients during treatment with BeneFIX.
• Outcomes:
  The primary outcomes are development of clinically significant FIX inhibitors, FIX hypersensitivity allergic reactions, thrombogenicity, lack of effect and red cell agglutination. Secondary safety outcomes include the frequency of AEs and SAEs,etc.Secondary efficacy outcomes will be:Annualized bleeding rates in subjects receiving treatment with BeneFIX,The responses to all on-demand treatment with BeneFIX for all bleeds,Number of BeneFIX infusions to treat each new bleed,Number of spontaneous/non traumatic breakthrough bleeds within 48 hours of a prophylaxis dose of BeneFIX
• Data analysis plan:
  All safety and efficacy analyses, except the analysis for Annualized Bleeding Rates(ABR),will be performed on all subjects who receive at least one dose of BeneFIX. ABR analysis will be performed on subjects who participated in prophylaxis period for at least 6 months. The efficacy results of this study will be presented using descriptive statistics. The following descriptive statistics will be used:1)For numeric endpoints: sample size, mean, standard deviation, median, minimum and maximum,2)For categorical endpoints: count and percentage.Safety data will be tabulated and listed according to Pfizer’s standard reporting algorithms. The reasons for discontinuation of BeneFIX therapy will be described.Subgroup analysis will be done by several sub-populations.

Documents

Study, other information

ANNEX 1 Subject Diary