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 GTC2638
Trial ID NCT04823611
Disease Hypercholesterolemia
Altered gene PCSK9
Therapeutic/Target gene Target gene
TherapyASO
Treatment AZD8233|IONIS-AZ4-2.5-LRx|ION449
PhasePhase1|Phase2
Recruitment statusCompleted
TitleA Phase 1 and 2 Study to Evaluate the Safety, Tolerability, Efficacy, Pharmacokinetics and Pharmacodynamics of AZD8233 Following a Multiple Subcutaneous Dose Administration in Japanese Participants With Dyslipidemia
Year2021
CountryJapan
Company sponsorAstraZeneca
Other ID(s)D7990C00006
Vector information
VectorGalNAc3

Clinical Result

Cohort1: AZD8233_dose level1
Administration route subcutaneous injection
Dosage AZD8233, 15 mg, Q4W, on Days 1, 29 and 57
Age Adult, Older_Adult
References PMID: 38504437
Cohort2: AZD8233_dose level2
Administration route subcutaneous injection
Dosage AZD8233, 50 mg, Q4W, on Days 1, 29 and 57
Age Adult, Older_Adult
References PMID: 38504437
Cohort3: AZD8233_dose level3
Administration route subcutaneous injection
Dosage AZD8233, 90 mg, Q4W, on Days 1, 8, 29 and 57
Age Adult, Older_Adult
References PMID: 38504437
Cohort4: Placebo
Administration route subcutaneous injection
Age Adult, Older_Adult
References PMID: 38504437

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