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 GTC0186
Trial ID NCT01961921
Disease 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
PhasePhase2
Recruitment statusCompleted
TitleA Phase 2, Multicenter, Open-Label, Extension Study to Evaluate the Long-Term Safety, Clinical Activity, and Pharmacokinetics of ALN-TTR02 in Patients With Familial Amyloidotic Polyneuropathy Who Have Previously Received ALN-TTR02
Year2013
CountryBrazil|France|Germany|Portugal|Spain|Sweden|United States
Company sponsorAlnylam Pharmaceuticals
Other ID(s)ALN-TTR02-003|2013-001644-65
Vector information
Vectorlipid nanoparticle

Clinical Result

Cohort 1
Administration route intravenous infusion
Pts 27
Age Adult, Older_Adult
Outcome The Number of Participants Experiencing Adverse Events (AEs), Serious Adverse Events (SAEs) and Study Drug Discontinuation:At least 1 Treatment Emergent Adverse Event (TAE) Measure Type: Number:26; At least 1 Serious Adverse Event (SAE):7; Study Drug Discontinuation for any reason:2
Adverse reactions 2/27(All-cause mortality); 7/27(Cardiac 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); Renal and urinary disorders; Surgical and medical procedures; Vascular disorders)
References PMID: 32641071

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