InformationClinical trials have complex eligibility criteria.
Always talk to your clinician about you’re interest in participating in a trial.
Learn why

Optimise reading forHealth ProfessionalsPatients

Closed (no longer recruiting)Last updated: 5 February 2024

This Phase III is evaluating the effectiveness of a targeted therapy (vutrisiran) in people with hereditary transthyretin amyloidosisHELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Clinical summary

Summary

Vutrisiran (ALN-TTRSC02) : Participants will receive vutrisiran during the Treatment and Treatment Extension Periods. Drug: Vutrisiran (ALN-TTRSC02) : Patisiran will be administered by intravenous (IV) infusion.

Conditions

This trial is treating patients with hereditary transthyretin amyloidosis (hATTR amyloidosis).

Cancer

Blood Cancers Haematological

Age

People18 - 85

Phase

III

More information

Trial Identifiers

Use the hyperlinks, where available to access additional clinical trial information.

Trial sponsor

Alnylam Pharmaceuticals

Scientific Title

HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Eligibility

Inclusion

  • Male or female of 18 to 85 years of age (inclusive);
  • Has a diagnosis of hATTR amyloidosis with transthyretin (TTR) mutation;
  • Has adequate neurologic impairment score (NIS);
  • Has adequate polyneuropathy disability (PND) score;
  • Has adequate Karnofsky Performance Status (KPS).

Exclusion

  • Had a prior liver transplant or is likely to undergo liver transplantation during the study;
  • Has known other (non-hATTR) forms of amyloidosis or leptomeningeal amyloidosis;
  • Has New York Heart Association heart failure classification >2;
  • Clinically significant liver function test abnormalities;
  • Has known human immunodeficiency virus (HIV), hepatitis C virus (HCV) or hepatitis B virus (HBV) infection;
  • Received an experimental drug within 30 days of dosing;
  • Received prior TTR-lowering treatment;
  • Has other known causes of neuropathy.

Exclusion

  • You have certain types of non-cancer medical conditions.
  • You have had certain treatments, surgical procedures or drugs.
Message

Clinical trials have complex eligibility criteria.

Ask your doctor if this trial could be right for you.

Participating hospitals

+ Show non-recruiting hospitals

Closed hospitals

InformationTell us if you find this trial availability is not accurate.Report inaccuracy

Get Support

Example

Cancer Connect

Speak with someone who has cancer clinical trial experience.

Learn more

Example

Cancer Council’s cancer nurses

If you need cancer information and practical support for yourself, a carer, family or friend, contact Cancer Council’s experienced cancer nurses on 131120.

Learn more

Example

Information for family, friends and carers

When you are considering a cancer clinical trial, it is a good idea to discuss it with your family, friends or carers.

Learn more

Victorian Cancer Registry Victorian Government

The Victorian Cancer Trials Link is supported by the Victorian Government through the Victorian Cancer Agency.

RAP

Cancer Council Victoria would like to acknowledge the traditional custodians of the land on which we live and work. We would also like to pay respect to the elders past and present and extend that respect to all other Aboriginal people.