A post-authorization study to assess the safety and efficacy of Fanhdi (Double-inactivated human anti-hemophilic factor) in subjects with Von Willebrand disease (PostAuthorization Study with Fanhdi in VWD patient)

Study type

• Study topic:
  Human medicinal product

  Disease /health condition
• Study type:
  Non-interventional study
• Scope of the study:
  Assessment of risk minimisation measure implementation or effectiveness

  Effectiveness study (incl. comparative)

  Safety study (incl. comparative)
• Data collection methods:
  Secondary data collection

Non-interventional study

• Non-interventional study design:
  Cohort

  Other
• Non-interventional study design, other:
  Phase 4, observational, multi-center, prospective, post-authorization study

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD06) von Willebrand factor and coagulation factor VIII in combination
• Medical condition to be studied:
  Von Willebrand's disease

Population studied

• Short description of the study population:
  Subjects aged 18 years or older diagnosed with Von Willebrand's disease (VWD) treated with Fanhdi under routine clinical practice.; Inclusion criteria:; 1. Male and female subjects ≥18 years of age diagnosed with hereditary VWD of any type and severity who require replacement therapy with VWD/FVIII concentrates when desmopressin (DDAVP) treatment alone is ineffective or contra-indicated.; 2. Subjects with a history of receiving prior treatment with VWF concentrates due to bleeding episodes and/or surgery or invasive procedures (on demand prophylaxis).; 3. Subjects who are expected to experience bleeding episodes and/or surgeries or invasive active bleeding at the time of inclusion.; 4. Subjects who are willing and able to provide written informed consent or have an authorized representative able to provide written informed consent on behalf of the subject in accordance with local law and institutional policy.; ; Exclusion criteria:; 1. Subjects diagnosed with acquired VWD.; 2. Subjects with a congenital or acquired platelet function disorder or other concomitant processes that may interfere with coagulation.; 3. Subjects who are positive for anti-VWF or anti-FVIII antibodies (≥0.5 Bethesda units) or has been positive in the history of their disease.; 4. Subjects with a known intolerance to any substance contained in Fanhdi.; 5. Subjects with a history of anaphylactic reactions to blood or blood components.; 6. Subjects who are participating in another clinical study involving an investigational treatment or have participated in one in the past 4 weeks.; 7. Subjects who, in the opinion of investigator, may have compliance problems with the protocol.
• Age groups:
  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:
  Other
• Special population of interest, other:
  Patients with Von Willebrand's disease
• Estimated number of subjects:
  15

Study design details

• Main study objective:
  To evaluate the safety (immunogenicity and thrombogenicity) associated with long term use of Fanhdi.
• Outcomes:
  * Adverse events including SAEs and suspected adverse drug reactions (ADRs) * Clinical laboratory values including inhibitor (immunogenicity) and functional activity testing * Thrombogenicity assessment * Vital signs * Physical examination, * Bleeding duration and severity * Investigator’s qualitative assessment of hemostasis * Amount of Fanhdi (IU/kg) used per subject, per year and per infusión * Use of other hemoderivatives per bleeding episode * Overall clinical efficacy
• Data analysis plan:
  The safety analyses will be addressed by listing and tabulation of AEs (includes suspected ADRs), clinical laboratory tests including inhibitor (immunogenicity) and functional activity testing, thrombogenicity, vital signs, and physical examinations. Data will be described using descriptive analyses.

Documents

Study results

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