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 GTC0470
Trial ID NCT03851705
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
TitleA Two-Part (Double-Blind Placebo Controlled/Open-Label) Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Subjects With Homozygous Familial Hypercholesterolemia (Hofh) (ORION-5)
Year2019
CountryChina|Israel|Russian Federation|Serbia|South Africa|Turkey|Ukraine
Company sponsorNovartis Pharmaceuticals
Other ID(s)MDCO-PCS-17-02|CKJX839A12302|2018-000893-31
Vector information
VectorGalNAc

Clinical Result

Cohort1: Inclisiran_Day 1 and Day 90
Administration route subcutaneous injection
Dosage Inclisiran sodium, 300 mg, Day 1 and Day 90
Pts 37
Age Adult, Older_Adult
Outcome Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150:2.39
Adverse reactions 0/37(All-cause mortality); 2/37(General disorders; Infections and infestations; Neoplasms benign, malignant and unspecified(incl cysts and polyps))
References PMID: 33990512
Cohort2: Placebo
Administration route subcutaneous injection
Dosage placebos, 300 mg, Day 1 and Day 90
Pts 19
Age Adult, Older_Adult
Outcome Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Day 150:0.70
Adverse reactions 0/19(All-cause mortality); 1/19(Social circumstances)
References PMID: 33990512
Cohort3: Inclisiran_Day 270, Day 450 and Day 630
Administration route subcutaneous injection
Dosage Inclisiran sodium, 300 mg, Day 270, Day 450 and Day 630
Pts 53
Age Adult, Older_Adult
Adverse reactions 3/53(All-cause mortality); 11/53(Cardiac disorders; General disorders; Hepatobiliary disorders; Infections and infestations; Injury, poisoning and procedural complications; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Renal and urinary disorders; Reproductive system and breast disorders; Respiratory, thoracic and mediastinal disorders)
References PMID: 33990512

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