HELIOS-A: A Phase 3 Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients with Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

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)

Status

Active, not recruiting

Phases

Phase 3

Study type

Interventional

Source

JPRN

Registry ID

JPRN-jRCT2080224769

Enrollment

Registered

2019-07-05

Start date

2019-07-31

Completion date

Unknown

Last updated

2026-06-29

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Hereditary Transthyretin Amyloidosis with polyneuropathy

Interventions

investigational material(s) Generic name etc : ALN-TTRSC02 INN of investigational material : vutrisiran Therapeutic category code : 129 Other agents affecting peripheral nervous system Dosage and Admi

Eligibility

Sex/Gender

All

Inclusion criteria

Inclusion criteria: -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 criteria

Exclusion criteria: -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

Design outcomes

Primary

Measure	Time frame
efficacy -Change from Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 [Time Frame: Baseline, Month 9] -Change from Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Total Score at Month 9 [Time Frame: Baseline, Month 9]	—

Secondary

Measure	Time frame
safety efficacy exploratory pharmacokinetics pharmacodynamics -Change from Baseline in the Timed 10-Meter Walk Test (10-MWT) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18] -Change from Baseline in the Modified Body Mass Index (mBMI) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ] -Change from Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ] -Percentage Reduction in Serum Transthyretin (TTR) Levels at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ] -Frequency of All-Cause Deaths and/or All-Cause Hospitalizations Up to Month 18 [ Time Frame: Up to Month 18 ] -Frequency of All-Cause Deaths and/or All-Cause Hospitalizations in Participants with Cardiac Involvement Up to Month 18 [ Time Frame: Up to Month 18 ]	—

Measure

Time frame

safety efficacy exploratory pharmacokinetics pharmacodynamics -Change from Baseline in the Timed 10-Meter Walk Test (10-MWT) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18] -Change from Baseline in the Modified Body Mass Index (mBMI) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ] -Change from Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ] -Percentage Reduction in Serum Transthyretin (TTR) Levels at Months 9 and 18 [ Time Frame: Baseline, Months 9 and 18 ] -Frequency of All-Cause Deaths and/or All-Cause Hospitalizations Up to Month 18 [ Time Frame: Up to Month 18 ] -Frequency of All-Cause Deaths and/or All-Cause Hospitalizations in Participants with Cardiac Involvement Up to Month 18 [ Time Frame: Up to Month 18 ]

—

Countries

Asia except Japan, Europe, Japan, North America, Oceania, South America

Outcome results

None listed

Source: JPRN (via WHO ICTRP) · Data processed: Jul 3, 2026