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 GTC1927
Trial ID NCT03593785
Disease Hypercholesterolemia
Altered gene PCSK9
Therapeutic/Target gene Target gene
TherapyASO
Treatment AZD8233|IONIS-AZ4-2.5-LRx|ION449
PhasePhase1
Recruitment statusCompleted
TitleA Phase I Randomized, Single-blind, Placebo-controlled Study to Assess the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AZD8233 Following Single Ascending Dose Administration to Healthy Male Subjects With Elevated LDL-C Levels.
Year2018
CountryUnited States
Company sponsorAstraZeneca
Other ID(s)D7990C00001
Vector information
VectorGalNAc3

Clinical Result

Cohort1: AZD8233_dose level1
Administration route subcutaneous injection
Dosage AZD8233, 4 mg, single dose, on Day 1
Age Adult
References PMID: 38504437
Cohort2: AZD8233_dose level2
Administration route subcutaneous injection
Dosage AZD8233, 12 mg, single dose, on Day 1
Age Adult
References PMID: 38504437
Cohort3: AZD8233_dose level3
Administration route subcutaneous injection
Dosage AZD8233, 20 mg, single dose, on Day 1
Age Adult
References PMID: 38504437
Cohort4: AZD8233_dose level4
Administration route subcutaneous injection
Dosage AZD8233, 30 mg, single dose, on Day 1
Age Adult
References PMID: 38504437
Cohort5: AZD8233_dose level5
Administration route subcutaneous injection
Dosage AZD8233, 60 mg, single dose, on Day 1
Age Adult
References PMID: 38504437
Cohort6: AZD8233_dose level6
Administration route subcutaneous injection
Dosage AZD8233, 90 mg, single dose, on Day 1
Age Adult
References PMID: 38504437
Cohort7: AZD8233_dose level7
Administration route subcutaneous injection
Dosage AZD8233, 120 mg, single dose, on Day 1
Age Adult
References PMID: 38504437
Cohort8: Placebo
Administration route subcutaneous injection
Age Adult
References PMID: 38504437

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