Basic Information

Gene symbol TTR Synonyms ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description transthyretin

GTO ID GTC2229
Trial ID NCT04153149
Disease Transthyretin Amyloidosis | Cardiomyopathy
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTRSC02|AMVUTTRA|Vutrisiran
Location approved US, EU, UK
PhasePhase3
Recruitment statusActive, Not Recruiting
TitleHELIOS-B: A Phase 3, Randomized, Double-blind, Placebo-controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Year2019
CountryArgentina|Australia|Austria|Belgium|Canada|Croatia|Czechia|Denmark|France|Germany|Hungary|Ireland|Israel|Japan|Korea, Republic of|Latvia|Lithuania|Netherlands|Norway|Peru|Poland|Portugal|Spain|Sweden|United Kingdom|United States
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTRSC02-003|2019-003153-28|2023-508366-15

Clinical Result

Cohort1: Vutrisiran
Administration route subcutaneous injection
Dosage Vutrisiran, 25 mg, q3M
Age Adult, Older_Adult
Cohort2: Placebo
Administration route subcutaneous injection
Age Adult, Older_Adult

Relationship Graph

Overview of Knowledge Graph