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 GTC0223
Trial ID NCT02292186
Disease Amyloidosis
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTRSC|revusiran
PhasePhase2
Recruitment statusCompleted
TitleA Phase 2, Open-label Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTRSC in Patients With Transthyretin (TTR) Cardiac Amyloidosis Who Have Previously Received ALN-TTRSC
Year2014
CountryUnited States|Canada|United Kingdom
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTRSC-003
Vector information
VectorGalNAc

Clinical Result

Cohort 1
Administration route None
Pts 25
Age Adult, Older_Adult
Outcome Safety and Tolerability Results of Long-term Dosing With ALN-TTRSC (Revusiran) Transthyretin (TTR) Cardiac Amyloidosis Patients:At least 1 AE (adverse event) Measure Type: Count of Participants:25 100.0%; At least 1 SAE (serious adverse event):22 88.0%; Study discontinuation for any reason:18 72.0% |Serum TTR Levels:Overall Number of Participants Analyzed:0
Adverse reactions 8/25(All-cause mortality); 22/25(Cardiac disorders; Gastrointestinal disorders; General disorders; Immune system 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; Respiratory, thoracic and mediastinal disorders; Vascular disorders)

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