Detail

Basic Information Clinical Results Relationship Graph

Basic Information

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	GTC0460
Trial ID	NCT03759379
Disease	Amyloidosis \| Transthyretin Amyloidosis
Altered gene	TTR
Therapeutic/Target gene	Target gene
Therapy	siRNA
Treatment	ALN-TTRSC02\|AMVUTTRA\|Vutrisiran;ALN-TTR02\|ONPATTRO\|patisiran
Location approved	US, EU, UK;US, EU, UK, Japan, Canada, Switzerland, Brazil, China, Israel, Turkey, Australia
Phase	Phase2
Recruitment status	Active, Not Recruiting
Title	HELIOS-A: A Phase 3 Global, Randomized, Open-label Study to Evaluate the Efficacy and Safety of ALN-TTRSC02 in Patients With Hereditary Transthyretin Amyloidosis (hATTR Amyloidosis)
Year	2019
Country	Argentina\|Australia\|Belgium\|Brazil\|Bulgaria\|Canada\|Cyprus\|France\|Germany\|Greece\|Italy\|Japan\|Korea,Republic of\|Malaysia\|Mexico\|Netherlands\|Portugal\|Spain\|Sweden\|China\|United Kingdom\|United States
Company sponsor	Alnylam Pharmaceuticals
Other ID(s)	ALN-TTRSC02-002\|2018-002098-23

Vector information

Vector	lipid nanoparticle\|ESC-GalNAc

Clinical Result

Cohort1: vutrisiran_vutrisiran

Administration route	subcutaneous injection
Dosage	vutrisiran, 25 mg, once every 3 months (q3M) for 18 months; vutrisiran, once every 6 months (q6M) or q3M
Pts	122
Age	Adult, Older_Adult
Outcome	Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348):Least Squares Mean (Standard Error):14.76(2.00)
Adverse reactions	2/122(All-cause mortality); 21/122(Cardiac disorders; Gastrointestinal disorders; General 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; Renal and urinary disorders; Vascular disorders)
References	PMID: 35875890

Cohort2: patisiran_vutrisiran

Administration route	intravenous injection\|subcutaneous injection
Dosage	patisiran, 0.3 mg/kg, once every 3 weeks (q3w) for 18 months; vutrisiran, q6M or q3M
Pts	42
Age	Adult, Older_Adult
Outcome	Change From Baseline in the Modified Neurologic Impairment Score +7 (mNIS+7) at Month 9 Between the Vutrisiran Group (HELIOS-A) and the External Placebo Comparator Group [APOLLO (NCT01960348):Least Squares Mean (Standard Error):-2.24(1.43)
Adverse reactions	3/42(All-cause mortality); 17/42(Cardiac disorders; Ear and labyrinth disorders; Gastrointestinal disorders; General disorders; Immune system disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Nervous system disorders; Renal and urinary disorders; Surgical and medical procedures; Vascular disorders)
References	PMID: 35875890

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