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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
Completed
Phases
Phase 3
Study type
Interventional
Source
EU CTIS
Registry ID
CTIS2023-508365-33-00
Acronym
ALN-TTRSC02-002
Enrollment
44
Registered
2024-06-12
Start date
2019-04-09
Completion date
2025-07-17
Last updated
2025-02-25

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

Conditions

Hereditary Transthyretin Amyloidosis

Brief summary

Change from baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) of the ALN-TTRSC02 group compared to the placebo arm of the Phase 3 patisiran-LNP study (ALN-TTR02-004; APOLLO)

Detailed description

Change from baseline in the following parameters of the ALN-TTRSC02 group compared to the placebo arm of the APOLLO (patisiran) study: - Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score - Timed 10-meter walk test (10-MWT) - mNIS+7 - 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 arm

Interventions

DRUGAmvuttra 25 mg solution for injection in pre-filled syringe
DRUGOnpattro 2 mg/mL concentrate for solution for infusion.

Sponsors

Alnylam Pharmaceuticals Inc., Alnylam Pharmaceuticals Inc., Alnylam Pharmaceuticals Inc.
Lead SponsorINDUSTRY

Eligibility

Sex/Gender
All
Age
18 Years to No maximum

Design outcomes

Primary

MeasureTime frame
Change from baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) of the ALN-TTRSC02 group compared to the placebo arm of the Phase 3 patisiran-LNP study (ALN-TTR02-004; APOLLO)—

Secondary

MeasureTime frame
Change from baseline in the following parameters of the ALN-TTRSC02 group compared to the placebo arm of the APOLLO (patisiran) study: - Norfolk Quality of Life-Diabetic Neuropathy (Norfolk QoL-DN) total score - Timed 10-meter walk test (10-MWT) - mNIS+7 - 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 arm—

Countries

Belgium, Bulgaria, Cyprus, Italy, Netherlands, Portugal, Spain

Outcome results

None listed

Source: EU CTIS · Data processed: Feb 4, 2026