TAK-660-403: Evaluation of Long-term Safety of ADYNOVI/ADYNOVATE (Antihaemophilic Factor [Recombinant] PEGylated, Rurioctocog Alfa Pegol) in Patients With Haemophilia A - An ADYNOVI/ADYNOVATE Post-Authorisation Safety Study (PASS) (TAK-660-403: ADYNOVI/ADYNOVATE PASS)

Study type

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

  Drug utilisation

Non-interventional study

• Non-interventional study design:
  Other
• Non-interventional study design, other:
  Prescription event monitoring, case-series

Study drug and medical condition

• Anatomical Therapeutic Chemical (ATC) code:
  (B02BD02) coagulation factor VIII

  coagulation factor VIII
• Medical condition to be studied:
  Haemophilia A without inhibitors

Population studied

• Age groups:
  Preterm newborn infants (0 – 27 days)

  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)
• Estimated number of subjects:
  200

Study design details

• Main study objective:
  The main aim of this study is to check for long-term side effects from ADYNOVI/ADYNOVATE prophylaxis in participants with haemophilia A when used under standard clinical practice in the real-world clinical setting.
• Outcomes:
  Incidence of Adverse Events (AE) and Serious Adverse Events (SAE). Occurrence of AE Related to: Impaired Renal Function, Impaired Hepatic Function, Impaired Neurologic Function. Change From Baseline at Specified Time Points in: Estimated Glomerular Filtration Rate (eGFR), Alanine Aminotransferase (ALT), Bilirubin, Polyethylene Glycol (PEG) Plasma Levels. Number of Participants With Clinically Significant Abnormalities in: Vital Signs, Physical Exam, Clinical Laboratory Parameters, Neurological Exam.
• Data analysis plan:
  All safety analyses for primary and secondary endpoints will be descriptive and, if applicable, be stratified by age group, etc. and may include arithmetic mean, standard deviation, median, quartiles and interquartile range, minimum, maximum, percentages, frequency counts, and 95% confidence intervals of select point estimates.