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 GTC0277
Trial ID NCT02597127
Disease Familial Hypercholesterolemia | Diabetes Mellitus | Arteriosclerotic Cardiovascular Disease
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 Compare the Effect of Different Doses of ALN-PCSSC Given as Single or Multiple Subcutaneous Injections in Subjects With High Cardiovascular Risk and Elevated LDL-C
Year2016
CountryCanada|Germany|Netherlands|United Kingdom|United States
Company sponsorThe Medicines Company
Other ID(s)MDCO-PCS-15-01
Vector information
VectorGalNAc

Clinical Result

Cohort1: ALN-PCSSC_dose level 1_bi-annual dosing
Administration route subcutaneous injection
Dosage ALN-PCSSC, 200mg, once at Day 1, bi-annual dosing
Pts 60
Age Adult, Older_Adult
Outcome percentage reduction from baseline in LDL cholesterol level at day 180: -27.9
Adverse reactions 0/60(All-cause mortality); 11/60(disorders; Gastrointestinal disorders; Injury, poisoning and procedural complications; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Renal and urinary disorders)
References PMID: 28306389 | 29735484 | 31553410
Cohort2: ALN-PCSSC_dose level 2_bi-annual dosing
Administration route subcutaneous injection
Dosage ALN-PCSSC, 300mg, once at Day 1, bi-annual dosing
Pts 61
Age Adult, Older_Adult
Outcome percentage reduction from baseline in LDL cholesterol level at day 180: -38.4
Adverse reactions 0/61(All-cause mortality); 11/61(Cardiac disorders; Gastrointestinal disorders; General disorders; Hepatobiliary disorders; Infections and infestations; Injury, poisoning and procedural complications; Investigations; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders)
References PMID: 28306389 | 29735484 | 31553410
Cohort3: ALN-PCSSC_dose level 3_bi-annual dosing
Administration route subcutaneous injection
Dosage ALN-PCSSC, 500mg, once at Day 1, bi-annual dosing
Pts 65
Age Adult, Older_Adult
Outcome percentage reduction from baseline in LDL cholesterol level at day 180: -41.9
Adverse reactions 1/65(All-cause mortality); 8/65(Blood and lymphatic system disorders; Cardiac disorders; Gastrointestinal disorders; General disorders; Injury, poisoning and procedural complications; Neoplasms benign, malignant and unspecified(incl cysts and polyps))
References PMID: 28306389 | 29735484 | 31553410
Cohort4: Normal Saline_bi-annual dosing
Administration route subcutaneous injection
Dosage Saline, once at Day 1, bi-annual dosing
Pts 65
Age Adult, Older_Adult
Outcome percentage reduction from baseline in LDL cholesterol level at day 180: 2.1
Adverse reactions 0/65(All-cause mortality); 3/65(Cardiac disorders; Injury, poisoning and procedural complications; Musculoskeletal and connective tissue disorders)
References PMID: 28306389 | 29735484 | 31553410
Cohort5: ALN-PCSSC_dose level 6_-quarterly dosing
Administration route subcutaneous injection
Dosage ALN-PCSSC, 100mg, once at Day 1 and Day 90, quarterly dosing
Pts 61
Age Adult, Older_Adult
Outcome percentage reduction from baseline in LDL cholesterol level at day 180: -35.5
Adverse reactions 0/61(All-cause mortality); 13/61(Cardiac disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Musculoskeletal and connective tissue disorders; Nervous system disorders; Vascular disorders)
References PMID: 28306389 | 29735484 | 31553410
Cohort6: ALN-PCSSC_dose level 1_-quarterly dosing
Administration route subcutaneous injection
Dosage ALN-PCSSC, 200mg, once at Day 1 and Day 90, quarterly dosing
Pts 62
Age Adult, Older_Adult
Outcome percentage reduction from baseline in LDL cholesterol level at day 180: -44.9
Adverse reactions 1/62(All-cause mortality); 8/62(Cardiac disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Renal and urinary disorders; Respiratory, thoracic and mediastinal disorders; Vascular disorders)
References PMID: 28306389 | 29735484 | 31553410
Cohort7: ALN-PCSSC_dose level 2_-quarterly dosing
Administration route subcutaneous injection
Dosage ALN-PCSSC, 300mg, once at Day 1 and Day 90, quarterly dosing
Pts 61
Age Adult, Older_Adult
Outcome percentage reduction from baseline in LDL cholesterol level at day 180: -52.6
Adverse reactions 0/61(All-cause mortality); 9/61(Cardiac disorders; Infections and infestations; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Vascular disorders)
References PMID: 28306389 | 29735484 | 31553410
Cohort8: Normal Saline_-quarterly dosing
Administration route subcutaneous injection
Dosage Saline, once at Day 1 and Day 90, quarterly dosing)
Pts 62
Age Adult, Older_Adult
Outcome percentage reduction from baseline in LDL cholesterol level at day 180: 1.8
Adverse reactions 0/62(All-cause mortality); 7/62(Cardiac disorders; Gastrointestinal disorders; Infections and infestations; Injury, poisoning and procedural complications; Investigations; Nervous system disorders; Renal and urinary disorders)
References PMID: 28306389 | 29735484 | 31553410

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