A Prospective, Non-interventional, Long-term, Multinational Cohort Safety Study of Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN) (TEG4001)

Study identification

• PURI:
  https://redirect.ema.europa.eu/resource/41080
• EU PAS number:
  EUPAS37728
• Study ID:
  41080
• Official title and acronym:
  A Prospective, Non-interventional, Long-term, Multinational Cohort Safety Study of Patients with Hereditary Transthyretin Amyloidosis with Polyneuropathy (hATTR-PN) (TEG4001)
• DARWIN EU® study:
  No
• Study countries:
  Austria

  Bulgaria

  Cyprus

  France

  Germany

  Greece

  Italy

  Portugal

  Spain

  United Kingdom
• Study description:
  This is a non-interventional, international, prospective cohort safety study of TEGSEDI-exposed and TEGSEDI-unexposed patients. Data will be extracted from medical records via secondary data collection. The TEGSEDI-exposed cohort will consist of patients diagnosed with hATTR-PN who are receiving TEGSEDI. Data from the TEGSEDI-exposed cohort will be compared to data collected prospectively from a TEGSEDI-unexposed cohort which will consist of patients diagnosed with hATTR-PN who have not taken any dose of TEGSEDI within 25 weeks prior to enrollment and are eligible for TEGSEDI treatment per the applicable product label. Among the patients enrolled in the TEGSEDI-exposed cohort, at least 96 adult subjects will meet the criteria of a “new user.” A new user is defined as a patient who has had no exposure to TEGSEDI prior to receiving commercial TEGSEDI. Patients in the TEGSEDI-unexposed cohort who begin TEGSEDI following enrollment will be analyzed as part of the TEGSEDI-exposed cohort from the time of initiation of TEGSEDI. The overarching goal of this study is to further characterize the long-term safety of TEGSEDI in patients with hATTR-PN under real-world conditions.
• Study status:
  Ongoing

Research institutions and networks

Institutions

Akcea Therapeutics, Inc.

Study timelines

Date when funding contract was signed

Planned: 16/12/2019

Actual: 16/12/2019

Study start date

Planned: 20/10/2021

Actual: 20/10/2021

Date of final study report

Planned: 30/06/2037

Sources of funding

Pharmaceutical company and other private sector 

More details on funding

Akcea Therapeutics, Inc.

Regulatory

• Was the study required by a regulatory body?:
  Yes
• Is the study required by a Risk Management Plan (RMP)?:
  EU RMP category 3 (required)

Other study registration identification numbers and links

TEG4001