Investigating Eplontersen PK in Plasma and Breast Milk in Healthy Lactating Participants

• Research type

  Research Study

• Full title

  An Open-label Study to Assess the Pharmacokinetics of Eplontersen in Plasma and Breast Milk Following Subcutaneous Administration of a Single Dose in Healthy Lactating Participants

• IRAS ID

  1007655

• Contact name

  Barry Reicher

• Contact email

  barry.reicher@astrazeneca.com

• Sponsor organisation

  AstraZeneca AB

• Research summary

  This clinical trial will recruit healthy, non-smoking, lactating female participants aged 18 years or older, who have already decided to stop breastfeeding and/or pump feeding permanently, and that decision was made independently of the decision to participate in this study. A total of approximately 12 healthy participants will take part in the clinical trial. The clinical trial will take place at one Clinical Unit in the UK and one Clinical Unit in the United States.

  The medicine to be tested in this trial is a compound called eplontersen, developed for a disease called transthyretin amyloidosis (ATTR). This disease is a progressive condition characterised by the buildup of abnormal transthyretin (TTR) protein deposits called amyloids that affect heart, bowels, and nerves.

  The main purpose of the clinical trial is to find how much of the clinical trial medicine is found in breast milk and blood after one dose is given by subcutaneous injection. The clinical trial will also investigate how the clinical trial medicine is taken up, metabolised (chemically broken down), distributed through the body and excreted (removed from the body). The clinical trial will also try to understand if the body develops a natural defence against the clinical trial medicine by producing antibodies against the clinical trial medicine (antibodies are proteins produced by the human body that help fight off foreign substances entering your body).

  The trial will comprise of:
  - Screening period of a maximum 28 days
  - In-house Treatment Period - admission on Day -1
  - Follow-up Visits on Day 8, Day 15, Day 29, Day 50, Day 71
  - An End of Study Visit on day 92 after the dose of clinical trial medication

  The clinical trial medicine, named eplontersen, has been already administered in healthy volunteers and patients as part of clinical trials and is now also approved in the UK. However, this medicine has not been administered in healthy lactating females yet.

• REC name

  North East - York Research Ethics Committee

• REC reference

  24/NE/0187

• Date of REC Opinion

  23 Jan 2025

• REC opinion

  Further Information Favourable Opinion