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 GTC2233
Trial ID NCT04155645
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 Multiple Ascending Subcutaneous Dose Administration in Subjects With Dyslipidemia With or Without Type 2 Diabetes
Year2019
CountryUnited States
Company sponsorAstraZeneca
Other ID(s)D7990C00002
Vector information
VectorGalNAc3

Clinical Result

Cohort1: AZD8233_dose level1
Administration route subcutaneous injection
Dosage AZD8233, 15 mg, Q4W, on Days 1, 8, 29 and 57
Age Adult, Older_Adult
References PMID: 38504437
Cohort2: AZD8233_dose level2
Administration route subcutaneous injection
Dosage AZD8233, 30 mg, Q4W, on Days 1, 8, 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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