Basic Information

Gene symbol PCSK9 Synonyms FH3, FHCL3, HCHOLA3, LDLCQ1, NARC-1, NARC1, PC9 Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas
Description proprotein convertase subtilisin/kexin type 9

GTO ID GTC0412
Trial ID NCT03399370
Disease Arteriosclerotic Cardiovascular Disease | Hypercholesterolemia
Altered gene PCSK9
Therapeutic/Target gene Target gene
TherapysiRNA
Treatment ALN-PCSSC|Inclisiran|KJX839|Leqvio
Location approved EU, UK, Australia, Canada, Israel, US, Saudi Arabia, Japan, China
PhasePhase3
Recruitment statusCompleted
TitleA Placebo-Controlled, Double-Blind, Randomized Trial to Evaluate the Effect of 300 mg of Inclisiran Sodium Given as Subcutaneous Injections in Subjects With Atherosclerotic Cardiovascular Disease (ASCVD) and Elevated Low-Density Lipoprotein Cholesterol (LDL-C)
Year2017
CountryUnited States
Company sponsorThe Medicines Company
Other ID(s)MDCO-PCS-17-04
Vector information
VectorGalNAc

Clinical Result

Cohort1: Inclisiran
Administration route subcutaneous injection
Dosage Inclisiran sodium, 300mg, Day 1, Day 90, then every 6 months
Pts 781
Age Adult, Older_Adult
Outcome Percentage Change in LDL-C From Baseline to Day 510:-56.34|Time-adjusted Percent Change in LDL-C Levels From Baseline After Day 90 and up to Day 540: -51.27
Adverse reactions 12/781(All-cause mortality); 175/781(Blood and lymphatic system disorders; Cardiac disorders; Endocrine 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)
References PMID: 32187462 | 37282500
Cohort2: Saline Solution
Administration route subcutaneous injection
Dosage Saline Solution, 300mg, Day 1, Day 90, then every 6 months
Pts 778
Age Adult, Older_Adult
Outcome Percentage Change in LDL-C From Baseline to Day 510:1.30|Time-adjusted Percent Change in LDL-C Levels From Baseline After Day 90 and up to Day 540: 2.51
Adverse reactions 11/778(All-cause mortality); 205/778(Blood and lymphatic system disorders; Cardiac disorders; Congenital, familial and genetic disorders; Eye disorders; Gastrointestinal disorders; General disorders; Hepatobiliary disorders; Immune system disorders; Infections and infestations; Injury, poisoning and procedural complications; Investigations; Metabolism and nutrition disorders; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders)
References PMID: 32187462 | 37282500

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