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Protocol NumberALN-TTRSC02-002
NCT Number(ClinicalTrials.gov Identfier)NCT03759379
Completed

2019-05-31 - 2026-12-31

Phase III

Recruiting3

ICD-10E85.0

Non-neuropathic heredofamilial amyloidosis

ICD-10E85.1

Neuropathic heredofamilial amyloidosis

ICD-10E85.2

Heredofamilial amyloidosis, unspecified

ICD-10E85.3

Secondary systemic amyloidosis

ICD-10E85.4

Organ-limited amyloidosis

ICD-10E85.8

Other amyloidosis

ICD-10E85.9

Amyloidosis, unspecified

ICD-9277.3

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)

  • Trial Applicant

    MEDPACE TAIWAN LIMITED

  • Sponsor

    Alnylam Pharmaceuticals, Inc.

  • Trial scale

    Multi-Regional Multi-Center

  • Update

    2026/02/01

Investigators and Locations

Principal Investigator Yi-Chun Lee Division of Neurology
Taipei Veterans General Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator Chi-Chao Chao Division of Neurology
National Taiwan University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Principal Investigator Long-Sun Ro Division of Neurology
Linkou Chang Gung Medical Foundation

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Recruiting

Condition/Disease

Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)

Objectives

Primary  To determine the efficacy of ALNTTRSC02 in patients with hATTR amyloidosis by evaluating the effect on neurologic impairment and on quality of life Secondary  To determine the efficacy of ALNTTRSC02 on gait speed, nutritional status, and disability  To characterize the effect of ALNTTRSC02 on serum TTR levels  To evaluate patient mortality and hospitalization Exploratory  To determine the effect of ALNTTRSC02 on:  Manifestations of cardiac amyloid involvement  Other assessment of neurologic impairment  Other assessments of quality of life  Disease stage  Performance of daily activities  To characterize the pharmacodynamic (PD) effect of ALN-TTRSC02 and patisiran-LNP on vitamin A levels  To characterize plasma pharmacokinetics (PK) of ALN-TTRSC02 and patisiran-LNP  To assess presence of antidrug antibodies (ADA) to ALN-TTRSC02 and patisiran-LNP

Test Drug

ALN-TTRSC02

Active Ingredient

ALN-65492

Dosage Form

injection

Dosage

50 mg/ml concentration; 25 mg/ 0.5 mL

Endpoints

Co-Primary:
 Change from baseline in the Modified
Neurologic Impairment Score +7
(mNIS+7) compared to the placebo arm
of the APOLLO study
 Change from baseline in Norfolk Quality
of Life-Diabetic Neuropathy (Norfolk
QoL-DN) total score compared to the
placebo arm of the APOLLO study

Secondary
 Change from baseline in the following
parameters compared to the placebo arm
of the APOLLO study:
 Timed 10-meter walk test (10-
MWT);
 Modified body mass index (mBMI)
 Rasch-built Overall Disability Scale
(R-ODS)
 Percent reduction in serum TTR levels in
the ALN-TTRSC02 arm compared to the
within-study patisiran-LNP arm
 Composite events of all-cause deaths
and/or all-cause hospitalizations in the
overall population (over 18 months)
compared to the placebo arm of the
APOLLO study
 Composite events of all-cause deaths
and/or all-cause hospitalizations in
patients with cardiac involvement (over
18 months) compared to patients with
cardiac involvement in the placebo arm of
the APOLLO study

Inclution 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.

The Estimated Number of Participants

  • Taiwan

    15 participants

  • Global

    160 participants

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