TRITON-PN

• Research type

  Research Study

• Full title

  TRITON-PN: A Phase 3, Global, Randomized, Open-Label Study to Evaluate the Efficacy and Safety of Nucresiran in Patients with Hereditary Transthyretin-Mediated Amyloidosis with Polyneuropathy (hATTR-PN)

• IRAS ID

  1013095

• Contact name

  Joana Radcliffe Stevens

• Contact email

  CRGUKStart-upteam.SM@thermofisher.com

• Sponsor organisation

  Alnylam Pharmaceuticals, Inc.

• Eudract number

  2025-522544-40

• Research summary

  hATTR amyloidosis is a rare genetic disease that tends to run in some families. hATTR amyloidosis is caused by certain variations in the gene for a protein called transthyretin, or TTR. The liver is the main organ that produces TTR protein, and TTR then circulates in the blood stream. Abnormal TTR protein can gradually deposit in many tissues and organs of the body, in collections of proteins called amyloid fibrils. These amyloid fibril collections can often affect the function of important organs such as the nerves, the heart, and the gut. This leads to symptoms of amyloidosis, including weakness, numbness, difficulty walking, shortness of breath, lightheadedness, and diarrhoea.

  Researchers are testing a new type of medicine called nucresiran. Nucresiran is an RNAi medicine. This means that the medicine
  works with the body to block or slow down the abnormal build-up of TTR protein. By lowering the amount of TTR protein in the body,
  researchers hope that nucresiran might slow down or stop the disease from getting worse.

  This clinical study aims to study the safety of nucresiran to find out what effects, if any, nucresiran has on people with hereditary transthyretin amyloidosis (hATTR) with polyneuropathy. The effects being studied include:

  • If the Study Drug, nucresiran, is able to improve neurological symptoms, quality of life, and physical function in patients with hATTR amyloidosis with polyneuropathy
  • If nucresiran is as effective as vutrisiran at lowering TTR protein in the blood

  Vutrisiran (developed by Alnylam) has been approved for use in hATTR patients with polyneuropathy by the Health Authority in the United States, Canada, Brazil, Switzerland, European Union, Japan, and Australia. Vutrisiran is also approved in the United Kingdom for the treatment of Hereditary transthyretin amyloidosis (hATTR). Like nucresiran, vutrisiran is designed to lower TTR protein levels in the body.

• REC name

  London - Westminster Research Ethics Committee

• REC reference

  25/LO/0798

• Date of REC Opinion

  24 Nov 2025

• REC opinion

  Further Information Favourable Opinion