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	GTC0185
Trial ID	NCT01960348
Disease	Familial Amyloid Neuropathy \| 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	APOLLO: A Phase 3 Multicenter, Multinational, Randomized, Double-blind, Placebo-controlled Study to Evaluate the Efficacy and Safety of Patisiran (ALN-TTR02) in Transthyretin (TTR)-Mediated Polyneuropathy (Familial Amyloidotic Polyneuropathy-FAP)
Year	2013
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-TTR02-004\|2013-002987-17

Vector information

Vector	lipid nanoparticle

Clinical Result

Cohort1: Patisiran

Administration route	intravenous infusion
Pts	148
Age	Adult, Older_Adult
Outcome	Modified Neuropathy Impairment Score +7 (mNIS+7):Least Squares Mean (Standard Error):-6.03(1.739)
Adverse reactions	7/148(All-cause mortality); 54/148(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; Renal and urinary disorders; Reproductive system and breast disorders; Respiratory, thoracic and mediastinal disorders; Skin and subcutaneous tissue disorders; Vascular disorders)
References	PMID: 31322739 \| 32131641 \| 28893208 \| 30878017 \| 36120830 \| 30586695 \| 29972753

Cohort2: Placebo

Administration route	intravenous infusion
Pts	77
Age	Adult, Older_Adult
Outcome	Modified Neuropathy Impairment Score +7 (mNIS+7):Least Squares Mean (Standard Error):27.96(2.602)
Adverse reactions	6/77(All-cause mortality); 31/77(Blood and lymphatic system disorders; Cardiac disorders; Congenital, familial and genetic 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; Respiratory, thoracic and mediastinal disorders; Skin and subcutaneous tissue disorders; Surgical and medical procedures; Vascular disorders)
References	PMID: 31322739 \| 32131641 \| 28893208 \| 30878017 \| 36120830 \| 30586695 \| 29972753

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