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 GTC0410
Trial ID NCT03397121
Disease Familial 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
TitlePlacebo-Controlled, Double-Blind, Randomized Trial to Evaluate the Effect of 300 mg of Inclisiran Sodium Given as Subcutaneous Injections in Subjects With Heterozygous Familial Hypercholesterolemia (HeFH) and Elevated Low-Density Lipoprotein Cholesterol (LDL-C).
Year2017
CountryCanada|Czechia|Denmark|Netherlands|South Africa|Spain|Sweden|United States
Company sponsorThe Medicines Company
Other ID(s)MDCO-PCS-17-03|2017-002472-30
Vector information
VectorGalNAc

Clinical Result

Cohort1: Inclisiran
Administration route subcutaneous injection|oral
Dosage Inclisiran sodium, 300mg, Day 1, Day 90, then every 6 months
Pts 242
Age Adult, Older_Adult
Outcome Percent Change in LDL-C From Baseline To Day 510:-41.15|Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 540:-38.08
Adverse reactions 1/241(All-cause mortality); 18/241(Cardiac disorders; Infections and infestations; Injury, poisoning and procedural complications; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Product Issues; Psychiatric disorders)
References PMID: 32197277
Cohort2: Placebo
Administration route subcutaneous injection|oral
Dosage Placebo, Day 1, Day 90 then every 6 months
Pts 240
Age Adult, Older_Adult
Outcome Percent Change in LDL-C From Baseline To Day 510:8.37|Time-adjusted Percent Change in LDL-C From Baseline After Day 90 and up to Day 540:6.22
Adverse reactions 1/240(All-cause mortality); 33/240(Cardiac disorders; Eye disorders; Gastrointestinal disorders; General disorders; Hepatobiliary disorders; Infections and infestations; Injury, poisoning and procedural complications; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Psychiatric disorders; Renal and urinary disorders; Respiratory, thoracic and mediastinal disorders)
References PMID: 32197277

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