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 GTC2217
Trial ID NCT04136171
Disease Transthyretin Amyloid Cardiomyopathy
Altered gene TTR
Therapeutic/Target gene Target gene
TherapyASO
Treatment AKCEA-TTR-LRx|Eplontersen|ION-682884|IONIS-TTR-LRx|ION-682884|Wainua
Location approved US
PhasePhase3
Recruitment statusActive, Not Recruiting
TitleA Phase 3 Global, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ION-682884 in Patients With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)
Year2020
CountryArgentina|Australia|Austria|Belgium|Brazil|Canada|Czechia|Denmark|France|Germany|Greece|Israel|Italy|Japan|Poland|Portugal|Puerto Rico|Spain|Sweden|United Kingdom|United States
Company sponsorIonis Pharmaceuticals, Inc.
Other ID(s)ION-682884-CS2|2019-002835-27
Vector information
VectorNo vector was used

Clinical Result

Cohort1: Eplontersen
Administration route subcutaneous injection
Dosage Eplontersen, once every 4 weeks
Age Adult, Older_Adult
Cohort2: Placebo
Administration route subcutaneous injection
Dosage placebo, once every 4 weeks
Age Adult, Older_Adult

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