Basic Information

Gene symbol APOA1 Synonyms AMYLD3, HPALP2, apo(a) Type of gene protein-coding
Gene perturbation-related omics dataset PerturbAtlas | PertOrg
Description apolipoprotein A1

GTO ID GTC2998
Trial ID NCT05337878
Disease Cardiovascular System Disease
Altered gene APOA1
Therapeutic/Target gene Target gene
TherapyASO
Treatment ISIS 681257|AKCEA-APO(a)-LRx|IONIS-APO(a)-LRx|TQJ230|Pelacarsen
Co-treatment Clopidogrel
PhasePhase1
Recruitment statusCompleted
TitleA Randomized Double-blind, Placebo-controlled, Non-confirmatory Study to Assess Safety, Tolerability, PK, and PD of Single Ascending and Multiple Doses of ISIS 681257 in Healthy Japanese Participants
Year2018
CountryUnited States
Company sponsorIonis Pharmaceuticals, Inc.
Other ID(s)AKCEA-CS1
Vector information
VectorGalNAc

Clinical Result

Cohort1: placebo_Single dose
Administration route subcutaneous injection
Dosage placebo, Single dose, on Day 1 of single-dose treatment period
Age Adult, Older_Adult
Cohort2: Pelacarsen_dose level1_single-dose treatment period
Administration route subcutaneous injection
Dosage Pelacarsen, 20 mg, Single dose, on Day 1 of single-dose treatment period
Age Adult, Older_Adult
Cohort3: Pelacarsen_dose level2_single-dose treatment period
Administration route subcutaneous injection
Dosage Pelacarsen, 40 mg, Single dose, on Day 1 of single-dose treatment period
Age Adult, Older_Adult
Cohort4: Pelacarsen_dose level3_single-dose treatment period
Administration route subcutaneous injection
Dosage Pelacarsen, 80 mg, Single dose, on Day 1 of single-dose treatment period
Age Adult, Older_Adult
Cohort5: placebo_Multiple doses
Administration route subcutaneous injection
Dosage placebo, Multiple doses, every 4 weeks, on Days 1, 29, 57 and 85 of multiple-dose treatment period
Age Adult, Older_Adult
Cohort6: Pelacarsen_dose level3_multiple-dose treatment period
Administration route subcutaneous injection
Dosage Pelacarsen, 80 mg, every 4 weeks, on Days 1, 29, 57 and 85 of multiple-dose treatment period
Age Adult, Older_Adult

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