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 GTC0228
Trial ID NCT02319005
Disease Amyloid Neuropathy | Transthyretin Amyloid Cardiomyopathy
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTRSC|revusiran
PhasePhase3
Recruitment statusCompleted
TitleA Phase 3 Multicenter, Multinational, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of ALN TTRSC in Patients With Transthyretin (TTR) Mediated Familial Amyloidotic Cardiomyopathy (FAC)
Year2014
CountryBelgium|Canada|France|Germany|Italy|Spain|Sweden|United Kingdom|United States
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTRSC-004
Vector information
VectorGalNAc

Clinical Result

Cohort1: ALN-TTRSC
Administration route subcutaneous injection
Pts 140
Age Adult, Older_Adult
Outcome 6 Minute Walk Distance (6-MWD):Overall Number of Participants Analyzed:0|Serum TTR Levels:Overall Number of Participants Analyzed:0
Adverse reactions 23/140(All-cause mortality); 83/140(Blood and lymphatic system disorders; Cardiac disorders; Ear and labyrinth disorders; Gastrointestinal disorders; General disorders; Hepatobiliary disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Psychiatric disorders; Renal and urinary disorders; Reproductive system and breast disorders; Respiratory, thoracic and mediastinal disorders; Vascular disorders)
References PMID: 32062791
Cohort2: Sterile Normal Saline
Administration route subcutaneous injection
Pts 66
Age Adult, Older_Adult
Outcome 6 Minute Walk Distance (6-MWD):Overall Number of Participants Analyzed:0|Serum TTR Levels:Overall Number of Participants Analyzed:0
Adverse reactions 7/66(All-cause mortality); 34/66(Blood and lymphatic system disorders; Cardiac disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Renal and urinary disorders; Respiratory, thoracic and mediastinal disorders; Skin and subcutaneous tissue disorders; Surgical and medical procedures; Vascular disorders)
References PMID: 32062791

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