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 GTC0275
Trial ID NCT02595983
Disease Transthyretin Amyloid Cardiomyopathy | Amyloid Neuropathy
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTRSC|revusiran
PhasePhase2
Recruitment statusCompleted
TitleAn Open-label Study to Evaluate the Efficacy and Safety of Revusiran in Patients With Transthyretin-mediated Familial Amyloidotic Polyneuropathy With Disease Progression Post-Orthotopic Liver Transplant
Year2015
CountryFrance|Germany|Portugal|Spain|Sweden|United Kingdom
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTRSC-005
Vector information
VectorGalNAc

Clinical Result

Cohort 1
Administration route subcutaneous injection
Dosage Revusiran, 500mg
Pts 12
Age Adult, Older_Adult
Outcome Percentage Change From Baseline in Serum TTR at Month 6:Mean (Standard Deviation):-72.0(18.39)
Adverse reactions 2/12(All-cause mortality); 8/12(Cardiac disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Psychiatric disorders; Renal and urinary disorders; Vascular disorders)

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