HELIOS-B

A phase 3, randomised, double-blind, placebo-controlled international study to evaluate the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with ATTR amyloidosis with cardiomyopathy.

Vutrisiran (ALN-TTRSC02) is a second generation RNA inhibitor TTR lowering drug, administered by subcutaneous injection. This trial will compare vutrisiran with placebo in patients with ATTR amyloidosis with cardiomyopathy.

What the study involves for patients

Participants will receive vutrisiran or placebo every 3 months over a period of 30-36 Months.

Who can take part in the trial

Patients diagnosed with either hereditary (hATTR) ATTR amyloidosis with cardiomyopathy, or wild-type (wtATTR) ATTR amyloidosis with cardiomyopathy,  

For a full list of inclusion and exclusion criteria, see the www.clinicaltrials.gov website.

Outcomes

The primary outcome assessed will be:

  • Composite endpoint of all-cause mortality and recurrent cardiovascular (CV) events.

Other outcomes assessed will include:

  • Change from baseline in the 6-minute walk test at month 30.
  • Change from baseline in the Kansas City Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) at month 30.
  • Change from baseline in mean left ventricular (LV) wall thickness by echocardiographic assessment at month 30.
  • Change from baseline in global longitudinal strain by echocardiographic assessment at month 30.
  • Composite endpoint of all-cause mortality and recurrent all-cause hospitalisations and urgent HF visits.
  • All-cause mortality.
  • Rate of recurrent CV events (CV hospitalisations and urgent HF visits).
  • Change from baseline in N-terminal prohormone B-type natriuretic peptide (NTproBNP) at month 30.

Timing

This study has completed recruitment and is ongoing at the NAC. Overall, 655 participants from around the world were enrolled including 151 NAC patients.