TAK-755-4007: A Post-Authorization Safety Study (PASS) to Further Evaluate Real-World Safety in Patients with Congenital Thrombotic Thrombocytopenic Purpura (cTTP) Treated with Adzynma

Study type

• Study topic:
  Disease /health condition

  Human medicinal product
• Study type:
  Non-interventional study
• Scope of the study:
  Safety study (incl. comparative)
• Data collection methods:
  Secondary use of data

Non-interventional study

• Non-interventional study design:
  Cohort

Study drug and medical condition

• Medicinal product name:
  ADZYNMA
• Study drug International non-proprietary name (INN) or common name:
  RADAMTS13
• Anatomical Therapeutic Chemical (ATC) code:
  (B01AD13) apadamtase alfa and cinaxadamtase alfa

  apadamtase alfa and cinaxadamtase alfa
• Medical condition to be studied:
  Congenital thrombotic thrombocytopenic purpura

Population studied

• Short description of the study population:
  Pediatric and adult participants who have received Adzynma for the treatment of cTTP, either prophylactically or as on-demand therapy for acute episodes-including those who became pregnant during Adzynma treatment.
• Age groups:
  • Paediatric Population (< 18 years)
  • Adult and elderly population (≥18 years)
• Estimated number of subjects:
  50

Study design details

• Study design:
  This non-interventional, retrospective, post-authorization safety, cohort study utilizes secondary data from medical records to evaluate the safety of Adzynma in real-world clinical practice.
• Main study objective:
  The main objective of this study is to learn more about the risk of children and adults with cTTP treated with Adzynma developing antibodies that prevent Adzynma from working properly (called neutralizing antibodies) within 6 months after the first treatment and to understand the risk of allergic reactions within 7 days of the first treatment with Adzynma.
• Outcomes:
  The primary outcomes will assess ; 1. Number of Participants With Hypersensitivity Reactions Following the Index Adzynma Infusions ; 2. Number of Participants With Neutralizing Antibodies to ADAMTS13 Following the Index Adzynma Infusions ; ; The secondary outcomes will assess ; 3. Number of Participants With Long Term Safety Risk of Hypersensitivity Reactions Following the Index Adzynma infusion ; 4. Number of Participants With Risk of Hypersensitivity Reactions After Each Administration of Adzynma Following the Index Infusion ; 5. Number of Participants With Treatment-emergent Adverse Events (TEAEs) ; 6. Gestational age at the Time of Infant Birth ; 7. Number of Participants With Spontaneous Abortion ; 8. Number of Participants With Stillbirth ; 9. Number of Participants With Induced Abortion ; 10. Number of Participants With Live Birth ; 11. Number of Participants With any Pregnancy Related Complications ; 12. Number of Participants Categorized According to Gestational age of Greater Than or Equal to (>=) 37 Gestational Weeks and Less Than (<) 37 Gestational Weeks at Birth ; 13. Number of Infants Categorized According to Year of Birth ; 14. Number of Infants With Normal Birth Weight ; 15. Number of Infants With Small for Gestational Age (SGA) ; 16. Number of Infants With Congenital Anomaly Detected at the Time of Birth ; 17. Number of Participants by Breastfeeding Status While Receiving Adzynma ; 18. Number of Infant Categorized by Growth and Development Outcomes.;
• Data analysis plan:
  The statistical analysis of the data will be primarily descriptive. Categorical variables will be presented as frequencies and percentages. Continuous variables will be presented as mean with standard deviation (SD) or standard error (SE) and range for normally distributed variables; and as mean and SD, median, interquartile range (IQR), and range for non-normally distributed variables.