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 GTC0554
Trial ID NCT04964557
Disease Hypercholesterolemia
Altered gene PCSK9
Therapeutic/Target gene Target gene
TherapyASO
Treatment AZD8233|IONIS-AZ4-2.5-LRx|ION449
PhasePhase2
Recruitment statusCompleted
TitleA Randomised, Parallel, Double-Blind, Placebo-Controlled Phase 2b Study to Assess the Safety, Tolerability and Efficacy of AZD8233 Treatment in Participants With Hyperlipidaemia
Year2021
CountryCzechia|Denmark|Hungary|Netherlands|Poland|Slovakia|Spain|United States
Company sponsorAstraZeneca
Other ID(s)D7990C00004|2020-005845-18
Vector information
VectorGalNAc3

Clinical Result

Cohort1: AZD8233
Administration route subcutaneous injection
Pts 207
Age Adult, Older_Adult
Adverse reactions 3/207(All-cause mortality); 18/207(Cardiac disorders; Ear and labyrinth disorders; Eye disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Psychiatric disorders; Respiratory, thoracic and mediastinal disorders)
Cohort2: Placebo
Administration route subcutaneous injection
Pts 203
Age Adult, Older_Adult
Adverse reactions 12/203(Cardiac disorders; Ear and labyrinth disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Psychiatric disorders; Vascular disorders)

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