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 GTC0211
Trial ID NCT02175004
Disease Familial Amyloid Polyneuropathy | Amyloidosis
Altered gene TTR
Therapeutic/Target gene Target gene
TherapyASO
Treatment ISIS 416858|IONIS-FXIRX|FXI-ASO|ISIS-FXI Rx|BAY2306001||BAY2306001|Fesomersen
PhasePhase3
Recruitment statusCompleted
TitleAn Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (FAP)
Year2014
CountryArgentina|Brazil|France|Germany|Italy|Portugal|Spain|United Kingdom|United States
Company sponsorIonis Pharmaceuticals, Inc.
Other ID(s)ISIS 420915-CS3|2013-004561-13
Vector information
VectorNo vector was used

Clinical Result

Cohort1: Previous Placebo
Administration route subcutaneous injection
Dosage Inotersen, 300 mg, weekly, up to 260 weeks
Pts 50
Age Adult, Older_Adult
Adverse reactions 2/50(All-cause mortality); 18/50(Cardiac disorders; Eye disorders; Gastrointestinal disorders; General disorders; Hepatobiliary disorders; Immune system disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Psychiatric disorders; Renal and urinary disorders; Reproductive system and breast disorders; Vascular disorders)
References PMID: 32343462 | 34355354 | 35799473 | 35908242
Cohort2: Previous Inotersen
Administration route subcutaneous injection
Dosage Inotersen, 300 mg, weekly, up to 260 weeks
Pts 85
Age Adult, Older_Adult
Adverse reactions 15/85(All-cause mortality); 46/85(Blood and lymphatic system disorders; Cardiac disorders; Gastrointestinal disorders; General disorders; Hepatobiliary 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; Renal and urinary disorders; Respiratory, thoracic and mediastinal disorders; Skin and subcutaneous tissue disorders; Vascular disorders)
References PMID: 32343462 | 34355354 | 35799473 | 35908242

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