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 GTC2328
Trial ID NCT04302064
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
PhasePhase1
Recruitment statusCompleted
TitleA Randomized, Double-Blinded, Placebo-Controlled, Study to Assess Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Single-Ascending and Multiple Doses of ION-682884 in Healthy Japanese Participants
Year2020
CountryUnited States
Company sponsorIonis Pharmaceuticals, Inc.
Other ID(s)ION-682884-CS20
Vector information
VectorNo vector was used

Clinical Result

Cohort1: Eplontersen_dose level 1
Administration route subcutaneous injection
Dosage Eplontersen, 45 mg
Pts 6
Age Adult, Older_Adult
References PMID: 35869634
Cohort2: Eplontersen_dose level 2
Administration route subcutaneous injection
Dosage Eplontersen, 60 mg
Pts 6
Age Adult, Older_Adult
References PMID: 35869634
Cohort3: Eplontersen_dose level 3
Administration route subcutaneous injection
Dosage Eplontersen, 90 mg
Pts 6
Age Adult, Older_Adult
References PMID: 35869634
Cohort4: Placebo
Administration route subcutaneous injection
Pts 6
Age Adult, Older_Adult
References PMID: 35869634

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