A Study to Evaluate Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy

• Research type

  Research Study

• Full title

  An Open-Label Extension Study to Assess the Safety and Efficacy of Vutrisiran in Patients with Transthyretin Amyloidosis with Cardiomyopathy (ATTR Amyloidosis with Cardiomyopathy)

• IRAS ID

  1010907

• Contact name

  Sasha Barrate

• Contact email

  UKStart-upteam.SM@ppd.com

• Sponsor organisation

  Alnylam Pharmaceuticals, Inc

• Research summary

  The purpose of this study is to test the safety of vutrisiran and find out what effects, if any, vutrisiran has on people with transthyretin amyloidosis (ATTR) with cardiomyopathy.
  ATTR amyloidosis is a rare and serious disease that occurs when abnormal forms of a protein called transthyretin (TTR) deposit throughout the body, including in the heart and nerves. When these abnormal proteins build up in the heart, they cause cardiomyopathy, where the heart muscle thickens and stiffens, affecting the heart’s ability to function properly.
  Vutrisiran is an approved therapy in some countries for the treatment of hereditary ATTR amyloidosis (hATTR) with polyneuropathy. However, it is considered to be an “investigational” medicine, meaning that it is not currently approved by regulatory authorities, including the Medicines and Healthcare products Regulatory Agency (MHRA) in the UK for the treatment of ATTR amyloidosis with cardiomyopathy.

  Participants will be invited to take part if they have participated in one of the parent studies (either Patisiran parent study ALN-TTR02-011 (APOLLO-B Open-Label Extension (OLE)) or ALN-TTRSC02-003 (HELIOS-B OLE). Participants will be in the study for up to 36 months and will visit the study site about 14 times during this period. During the treatment period, the study healthcare team will perform standard tests that include:
  • Collecting blood samples, (approx. 23 teaspoons across 36 months)
  • Collecting urine samples (if applicable),
  • Questionnaires.
  At the study site, participants will also receive 25 mg of vutrisiran at their appointments every 3 months. Study Drug will be given as an injection under the skin in the abdomen, upper arms, or thighs.

  This study will include approximately 7 sites in the UK consisting of NHS and non-NHS sites.

• REC name

  London - Surrey Borders Research Ethics Committee

• REC reference

  24/LO/0858

• Date of REC Opinion

  16 Dec 2024

• REC opinion

  Further Information Favourable Opinion