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 identification

• EU PAS number:
  EUPAS1000000870
• Study ID:
  1000000870
• Official title and acronym:
  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
• DARWIN EU® study:
  No
• Study countries:
  Austria

  France

  Germany

  Italy

  Poland

  Spain

  Switzerland

  United Kingdom
• Study description:
  Congenital thrombotic thrombocytopenic purpura (cTTP) is a rare blood disorder that some people are born with. It is caused by a problem in a gene passed down from parents to children, which affects the body’s ability to produce a enzyme called ADAMTS13. This enzyme helps to cut down a larger form of protein called von Willebrand Factor (VWF). People with cTTP have low levels of ADAMTS13. Without ADAMTS13, large forms of vWF build up and cause blood clots in small blood vessels. These clots can block blood flow to vital organs, causing serious health problems. Adzynma is a human ADAMTS13 protein made in the laboratory. It works the same way as natural ADAMTS13 does and may provide higher levels of ADAMTS13.; ; The main aim 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. Other aims are to better understand how safe treatment with Adzynma is over a longer period of time (called long-term safety) in children and adults with cTTP and to gather information about pregnancies and babies of women who have received Adzynma while pregnant.; ; Only data already available in the medical records of the people who received Adzynma for the treatment of cTTP in normal clinical practice will be reviewed and collected during this study.; Only data already available in the medical records of the participants who received Adzynma for the treatment of cTTP will be reviewed and collected during this study.
• Study status:
  Planned

Study timelines

Date when funding contract was signed

Planned:13/09/2024

Actual:13/09/2024

Study start date

Planned:30/04/2026

Date of final study report

Planned:01/12/2030

Sources of funding

More details on funding

Takeda

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 2 (specific obligation of marketing authorisation)