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 GTC0159
Trial ID NCT01737398
Disease Amyloidosis | Familial Amyloid Polyneuropathy
Altered gene TTR
Therapeutic/Target gene Target gene
TherapyASO
Treatment ISIS 416858|IONIS-FXIRX|FXI-ASO|ISIS-FXI Rx|BAY2306001||BAY2306001|Fesomersen
PhasePhase2|Phase3
Recruitment statusCompleted
TitleA Phase 2/3 Randomized, Double-Blind, Placebo-Controlled Study to Assess the Efficacy and Safety of ISIS 420915 in Patients With Familial Amyloid Polyneuropathy (NEURO-TTR Study)
Year2012
CountryArgentina|Brazil|France|Germany|Italy|New Zealand|Portugal|Spain|United Kingdom|United States
Company sponsorIonis Pharmaceuticals, Inc.
Other ID(s)ISIS 420915-CS2|2012-001831-30
Vector information
VectorNo vector was used

Clinical Result

Cohort1: Inotersen
Administration route subcutaneous injection
Dosage 300 mg
Pts 85
Age Adult, Older_Adult
Outcome At week 66, the changes from baseline were as follows: the Modified Neuropathy Impairment Score (mNIS) +7 Composite Score increased by 4.16 (standard deviation: 15.672); the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire score decreased by 0.08 (standard deviation: 18.967); the Norfolk QoL-DN Questionnaire Symptoms Domain Score decreased by 1.40 (standard deviation: 4.763); and the Norfolk QoL-DN Questionnaire Physical Functioning/Large Fiber Neuropathy Domain Score increased by 1.05 (standard deviation: 11.924).
Adverse reactions 5/112(All-cause mortality); 36/112(Blood and lymphatic system disorders; Cardiac disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Nervous system disorders; Psychiatric disorders; Renal and urinary disorders; Respiratory, thoracic and mediastinal disorders; Vascular disorders)
References PMID: 29972757 | 31853709
Cohort2: placebo
Administration route subcutaneous injection
Dosage Placebo
Pts 85
Age Adult, Older_Adult
Outcome At week 66, the changes from baseline were as follows: the Modified Neuropathy Impairment Score (mNIS) +7 Composite Score increased by 23.89 (standard deviation: 24.190); the Norfolk Quality of Life Diabetic Neuropathy (QoL-DN) Questionnaire score increased by 10.77 (standard deviation: 21.134); the Norfolk QoL-DN Questionnaire Symptoms Domain Score increased by 1.18 (standard deviation: 5.270); and the Norfolk QoL-DN Questionnaire Physical Functioning/Large Fiber Neuropathy Domain Score increased by 8.74 (standard deviation: 9.689).
Adverse reactions 13/60(Cardiac disorders; Gastrointestinal disorders; Infections and infestations; Injury, poisoning and procedural complications; Nervous system disorders; Vascular disorders)
References PMID: 29972757 | 31853709

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