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 GTC0539
Trial ID NCT04666298
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
PhasePhase2
Recruitment statusCompleted
TitleA Placebo-controlled, Double-blind, Randomized Trial to Evaluate the Effect of Different Doses of Inclisiran Given as Subcutaneous Injections in Japanese Participants With High Cardiovascular Risk and Elevated LDL-C
Year2021
CountryJapan
Company sponsorNovartis Pharmaceuticals
Other ID(s)CKJX839A11201
Vector information
VectorGalNAc

Clinical Result

Cohort1: Inclisiran_dose level 1
Administration route subcutaneous injection
Dosage Inclisiran sodium, 300 mg, Day 1, 90 and 270
Pts 57
Age Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) to Day 180:-56.3
Adverse reactions 0/99(All-cause mortality); 7/99(Cardiac disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Metabolism and nutrition disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps))
References PMID: 33990512
Cohort2: Inclisiran_dose level 2
Administration route subcutaneous injection
Dosage Inclisiran sodium, 200 mg, Day 1, 90 and 270
Pts 55
Age Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) to Day 180:-51.9
Adverse reactions 0/101(All-cause mortality); 7/101(Cardiac disorders; Eye disorders; Infections and infestations; Metabolism and nutrition disorders; Musculoskeletal and connective tissue disorders; Vascular disorders)
References PMID: 33990512
Cohort3: Inclisiran_dose level 3
Administration route subcutaneous injection
Dosage Inclisiran sodium, 100 mg, Day 1, 90 and 270
Pts 101
Age Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) to Day 180:-47.6
Adverse reactions 0/55(All-cause mortality); 2/55(Cardiac disorders; Injury, poisoning and procedural complications)
References PMID: 33990512
Cohort4: Placebo
Administration route subcutaneous injection
Dosage Placebo, Day 1, 90 and 270
Pts 99
Age Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) to Day 180:9.0
Adverse reactions 1/57(All-cause mortality); 6/57(Cardiac disorders; Eye disorders; General disorders; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders)
References PMID: 33990512

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