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 GTC0537
Trial ID NCT04641299
Disease Hypercholesterolemia
Altered gene PCSK9
Therapeutic/Target gene Target gene
TherapyASO
Treatment AZD8233|IONIS-AZ4-2.5-LRx|ION449
PhasePhase2
Recruitment statusCompleted
TitleA Randomized, Parallel, Double-blind, Placebo-controlled, Dose-ranging, Phase 2b Study to Evaluate the Efficacy, Safety and Tolerability of AZD8233 Treatment in Participants With Dyslipidemia
Year2020
CountryUnited States|Denmark|Slovakia
Company sponsorAstraZeneca
Other ID(s)D7990C00003
Vector information
VectorGalNAc3

Clinical Result

Cohort1: Placebo
Administration route subcutaneous injection
Pts 29
Age Adult, Older_Adult
Outcome Change in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12:Geometric Least Squares Mean (95% Confidence Interval):0.978(0.834 to 1.147)
Adverse reactions 0/29(All-cause mortality); 1/29(Injury, poisoning and procedural complications)
Cohort2: AZD8233_dose level1
Administration route subcutaneous injection
Dosage AZD8233, 15 mg, Q4W, on Days 1, 8, 29 and 57
Pts 30
Age Adult, Older_Adult
Outcome Change in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12:Geometric Least Squares Mean (95% Confidence Interval):0.206(0.174 to 0.242)
Adverse reactions 0/30(All-cause mortality); 2/30(Cardiac disorders; Infections and infestations; Respiratory, thoracic and mediastinal disorders; Vascular disorders)
Cohort3: AZD8233_dose level2
Administration route subcutaneous injection
Dosage AZD8233, 50 mg, Q4W, on Days 1, 8, 29 and 57
Pts 30
Age Adult, Older_Adult
Outcome Change in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12:Geometric Least Squares Mean (95% Confidence Interval):0.270(0.230 to 0.317)
Adverse reactions 0/30(All-cause mortality); 1/30(Cardiac disorders)
Cohort4: AZD8233_dose level3
Administration route subcutaneous injection
Dosage AZD8233, 90 mg, Q4W, on Days 1, 8, 29 and 57
Pts 30
Age Adult, Older_Adult
Outcome Change in Low-density Lipoprotein Cholesterol (LDL-C) at Week 12:Geometric Least Squares Mean (95% Confidence Interval):0.606(0.517 to 0.710)
Adverse reactions 0/30(All-cause mortality); 2/30(Cardiac disorders; Infections and infestations; Respiratory, thoracic and mediastinal disorders; Vascular disorders)

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