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 GTC0491
Trial ID NCT03997383
Disease Transthyretin Amyloidosis
Altered gene TTR
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-TTR02|ONPATTRO|patisiran
Location approved US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia
PhasePhase3
Recruitment statusActive, Not Recruiting
TitleAPOLLO-B: A Phase 3, Randomized, Double-blind, Placebo-controlled Multicenter Study to Evaluate the Efficacy and Safety of Patisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)
Year2019
CountryArgentina|Australia|Belgium|Brazil|Bulgaria|Chile|Czechia|Denmark|France|China|Italy|Japan|Korea,Republic of|Mexico|Netherlands|New Zealand|Portugal|Sweden|United Kingdom|United States
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTR02-011|2019-001458-24
Vector information
Vectorlipid nanoparticle

Clinical Result

Cohort1: Placebo
Administration route intravenous infusion
Dosage placebo, every 3 weeks during the 12-month double-blind (DB) treatment period
Pts 178
Age Adult, Older_Adult
Adverse reactions 8/178(All-cause mortality); 63/178(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)
References PMID: 37888916
Cohort2: Patisiran_DB treatment period
Administration route intravenous infusion
Dosage patisiran, 0.3 mg/kg every 3 weeks during the 12-month DB treatment period
Pts 181
Age Adult, Older_Adult
Adverse reactions 4/181(All-cause mortality); 61/181(Blood and lymphatic system disorders; Cardiac disorders; Gastrointestinal disorders; General disorders; Hepatobiliary disorders; Immune system disorders; Infections and infestations; Injury, poisoning and procedural complications; 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; Surgical and medical procedures)
References PMID: 37888916
Cohort3: Placebo (in DB Period)_Patisiran
Administration route intravenous infusion
Dosage patisiran, 0.3 mg/kg, every 3 weeks during the 36-month open label extension (OLE) period
Pts 166
Age Adult, Older_Adult
Adverse reactions 10/166(All-cause mortality)
References PMID: 37888916
Cohort4: Patisiran (in DB Period)_Patisiran
Administration route intravenous infusion
Dosage patisiran, 0.3 mg/kg, every 3 weeks during the 36-month OLE period
Pts 168
Age Adult, Older_Adult
Adverse reactions 9/168(All-cause mortality)
References PMID: 37888916

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