241302: POST-MARKETING non-interventional safety evaluation of obizur in the treatment of bleeding episodes for patients with acquired hemophilia A (241302: US Post-Marketing Safety Study)

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

  Drug utilisation

  Effectiveness study (incl. comparative)

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

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prospective and retrospective post-marketing safety surveillance study

Study drug and medical condition

• Study drug International non-proprietary name (INN) or common name:
  ANTIHEMOPHILIC FACTOR (RECOMBINANT) FORMULATED WITH SUCROSE
• Medical condition to be studied:
  Acquired haemophilia

Population studied

• Short description of the study population:
  Patients with acquired hemophilia A (AHA) were eligible for participation in this study if they were treated with Obizur in a hospital setting. All patients were required to be prescribed Obizur by a physician, prior to the decision to enroll in the study.; ; Inclusion Criteria; Subjects who meet ALL of the following criteria are eligible for this study:; 1. Subject is ≥18 years of age at the time of informed consent.; 2. Subject has AHA, and is being treated/was treated with Obizur.; 3. Subject or subject’s legally authorized representitive is willing and able to provide informed consent, unless informed consent is not required (e.g., deceased subjects, as local regulations allow).; ; Exclusion Criteria; Subjects who meet ANY of the following criteria are not eligible for this study:; 1. Subject has a known anaphylactic reaction to the active substance, to any of the excipients, or to hamster protein.; 2. Subject has a concomitant bleeding disorder(s) other than AHA.; 3. Subject has participated in another clinical study involving a medicinal product or device within 30 days prior to enrollment or is scheduled to participate in another clinical study involving a medicinal product or device during the course of the study.
• 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:
  Acquired hemophilia A patients
• Estimated number of subjects:
  53

Study design details

• Main study objective:
  The primary objective is to determine the incidence of therapy-related serious adverse events (SAEs) in patients with AHA who are prescribed and treated with Obizur in routine clinical practice.
• Outcomes:
  Incidence of therapy-related Serious Adverse Events (SAEs), Hemostatic effectiveness assessment for resolution of bleeding, Time to bleeding resolution, participant study termination, or switch to another treatment, Number of Obizur units/kg and infusions required for control of bleeding, Titer of anti-pFVIII inhibitors, Impact of inhibitor on hemostatic efficacy, Occurrence of hypersensitivity reactions, Occurrence of any thrombogenic event
• Data analysis plan:
  SAEs will be described in listings and tables, and incidence rates will be calculated. Tables outlining the hemostatic effectiveness and immunogenicity of Obizur, frequency, total dose, and total number of infusions of Obizur required to control bleeding episodes will be developed.