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 GTC0580
Trial ID 2013-004561-13
Disease 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
PhasePhase3
Recruitment statusCompleted
TitleAn Open-Label Extension Study to Assess the Long-Term Safety and Efficacy of ISIS 420915 in Patients with Familial Amyloid Polyneuropathy (FAP)
Year2014
CountryPortugal
Company sponsorIonis Pharmaceuticals, Inc
Other ID(s)NCT02175004|ISIS 420915-CS3
Vector information
VectorNo vector was used

Clinical Result

Cohort 1
Administration route subcutaneous injection
Dosage 300 mg
Pts 85
Age Adult, Older_Adult
Outcome 47.1 percent of participants decreased of >=7% from Baseline in weight.11.8 percent of participants increased of >=7% from Baseline in weight.
Adverse reactions 15/85(All-cause mortality); 46/85(Blood and lymphatic system disorders; Cardiac disorders; Eye disorders; Gastrointestinal disorders; General disorders; Hepatobiliary disorders; Immune system 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; Respiratory, thoracic and mediastinal disorders; Skin and subcutaneous tissue disorders; Vascular disorders)

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