Detail

Basic Information Clinical Results Relationship Graph

Gene symbol	TTR	Synonyms	ATTR, CTS, CTS1, HEL111, HsT2651, PALB, TBPA, TTN	Type of gene	protein-coding
Chromosome	18	Map location	18q12.1	dbXrefs
Gene perturbation-related omics dataset	PerturbAtlas
Description	transthyretin

GTO ID	GTC0253
Trial ID	NCT02510261
Disease	Amyloidosis
Altered gene	TTR
Therapeutic/Target gene	Target gene
Therapy	siRNA
Treatment	ALN-TTR02\|ONPATTRO\|patisiran
Location approved	US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia
Phase	Phase3
Recruitment status	Completed
Title	A Multicenter, Open-Label, Extension Study to Evaluate the Long-term Safety and Efficacy of Patisiran in Patients With Familial Amyloidotic Polyneuropathy Who Have Completed a Prior Patisiran Clinical Study
Year	2015
Country	Argentina\|Australia\|Brazil\|Bulgaria\|Canada\|Cyprus\|France\|Germany\|Italy\|Japan\|Korea,Republic of\|Malaysia\|Mexico\|Netherlands\|Portugal\|Spain\|Sweden\|China\|Turkey\|United Kingdom\|United States
Company sponsor	Alnylam Pharmaceuticals
Other ID(s)	ALN-TTR02-006\|2014-003877-40

Vector information

Vector	lipid nanoparticle

Cohort1: Prior Placebo Group of Study 004

Administration route	intravenous infusion
Dosage	patisiran, 0.3 mg/kg, once every 3 weeks (Q3W) up to 65.5 months
Pts	49
Age	Adult, Older_Adult
Outcome	Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation:Measure Type: Number:49.0
Adverse reactions	19/49(All-cause mortality); 39/49(Cardiac disorders; Congenital, familial and genetic disorders; Endocrine disorders; Eye 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; Reproductive system and breast disorders; Respiratory, thoracic and mediastinal disorders; Skin and subcutaneous tissue disorders; Social circumstances; Vascular disorders)
References	PMID: 33212063

Cohort2: Prior Patisiran Group of Study 004_up to 66.9 months

Administration route	intravenous infusion
Dosage	patisiran, 0.3 mg/kg, once every 3 weeks (Q3W) up to 66.9 months
Pts	137
Age	Adult, Older_Adult
Outcome	Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation:Measure Type: Number:16.8
Adverse reactions	21/137(All-cause mortality); 82/137(Blood and lymphatic system disorders; Cardiac disorders; Congenital, familial and genetic disorders; Ear and labyrinth disorders; Eye 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; Product Issues; Renal and urinary disorders; Reproductive system and breast disorders; Respiratory, thoracic and mediastinal disorders; Social circumstances; Vascular disorders)
References	PMID: 33212063

Cohort3: Prior Patisiran Group of Study 004_up to 61.4 months

Administration route	intravenous infusion
Dosage	patisiran, 0.3 mg/kg, once every 3 weeks (Q3W) up to 61.4 months
Pts	25
Age	Adult, Older_Adult
Outcome	Percentage of Participants With Adverse Events (AEs) Leading to Study Discontinuation:Measure Type: Number:0.0
Adverse reactions	1/25(All-cause mortality); 12/25(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; Psychiatric disorders; Renal and urinary disorders)
References	PMID: 33212063

Overview of Knowledge Graph