HELIOS-A: A Study of Vutrisiran (ALN-TTRSC02) in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
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ClinicalTrials.gov Identifier: NCT03759379 |
Recruitment Status :
Active, not recruiting
First Posted : November 30, 2018
Last Update Posted : February 11, 2021
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Condition or disease | Intervention/treatment | Phase |
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Amyloidosis, Hereditary Transthyretin Amyloidosis | Drug: Patisiran Drug: Vutrisiran (ALN-TTRSC02) | Phase 3 |
Study Type : | Interventional (Clinical Trial) |
Actual Enrollment : | 164 participants |
Allocation: | Randomized |
Intervention Model: | Parallel Assignment |
Masking: | None (Open Label) |
Primary Purpose: | Treatment |
Official 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) |
Actual Study Start Date : | February 14, 2019 |
Actual Primary Completion Date : | November 10, 2020 |
Estimated Study Completion Date : | May 2024 |

Arm | Intervention/treatment |
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Experimental: Vutrisiran (ALN-TTRSC02)
Participants will receive vutrisiran during the Treatment and Treatment Extension Periods.
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Drug: Vutrisiran (ALN-TTRSC02)
Vutrisiran will be administered by subcutaneous (SC) injection. |
Active Comparator: Patisiran
Participants will receive patisiran during the Treatment Period and will switch to vutrisiran during the Treatment Extension Period.
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Drug: Patisiran
Patisiran will be administered by intravenous (IV) infusion. Drug: Vutrisiran (ALN-TTRSC02) Vutrisiran will be administered by subcutaneous (SC) injection. |
- Change from Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 [ Time Frame: Baseline, Month 9 ]The mNIS+7 is a composite score that quantifies motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
- Change from Baseline in Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) Total Score at Month 9 [ Time Frame: Baseline, Month 9 ]The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.
- Change from Baseline in the Timed 10-Meter Walk Test (10-MWT) at Month 9 [ Time Frame: Baseline, Month 9 ]
- Change from Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 18 [ Time Frame: Baseline, Month 18 ]The mNIS+7 is a composite score that quantifies motor, sensory, and autonomic neurologic impairment due to injury of large and small nerves. The minimum and maximum values are 0 and 304, respectively. A higher score indicates a worse outcome.
- Change from Baseline in Norfolk QoL-DN Total Score at Month 18 [ Time Frame: Baseline, Month 18 ]The Norfolk QoL-DN questionnaire is a standardized 35-item patient-reported outcomes measure that is sensitive to the different features of diabetic neuropathy - small fiber, large fiber, and autonomic nerve function. The minimum and maximum values are -4 and 136, respectively. A higher score indicates a worse outcome.
- Change from Baseline in the 10-MWT at Month 18 [ Time Frame: Baseline, Month 18 ]
- Change from Baseline in the Modified Body Mass Index (mBMI) at Month 18 [ Time Frame: Baseline, Month 18 ]
- Change from Baseline in the Rasch-Built Overall Disability Scale (R-ODS) at Month 18 [ Time Frame: Baseline, Month 18 ]The R-ODS is comprised of a 24-item linearly weighted scale that specifically captures activity and social participation limitations. The minimum and maximum values are 0 and 48, respectively. A higher score indicates a better outcome.
- Percentage Reduction in Serum Transthyretin (TTR) Levels Through Month 18 [ Time Frame: Up to Month 18 ]

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Ages Eligible for Study: | 18 Years to 85 Years (Adult, Older Adult) |
Sexes Eligible for Study: | All |
Accepts Healthy Volunteers: | No |
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:
- 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.

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03759379

Study Director: | Medical Director | Alnylam Pharmaceuticals |
Responsible Party: | Alnylam Pharmaceuticals |
ClinicalTrials.gov Identifier: | NCT03759379 |
Other Study ID Numbers: |
ALN-TTRSC02-002 2018-002098-23 ( EudraCT Number ) |
First Posted: | November 30, 2018 Key Record Dates |
Last Update Posted: | February 11, 2021 |
Last Verified: | February 2021 |
Individual Participant Data (IPD) Sharing Statement: | |
Plan to Share IPD: | No |
Studies a U.S. FDA-regulated Drug Product: | Yes |
Studies a U.S. FDA-regulated Device Product: | No |
Amyloid Neuropathies, Familial Amyloidosis, Familial Amyloidosis Proteostasis Deficiencies Metabolic Diseases Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases |
Nervous System Diseases Amyloid Neuropathies Peripheral Nervous System Diseases Neuromuscular Diseases Genetic Diseases, Inborn Metabolism, Inborn Errors |