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 GTC0361
Trial ID NCT03070782
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
PhasePhase2
Recruitment statusCompleted
TitleA Randomized, Double-blind, Placebo-Controlled, Dose-Ranging Phase 2 Study of ISIS 681257 (AKCEA-APO(a)-LRx) Administered Subcutaneously to Patients With Hyperlipoproteinemia(a) and Established Cardiovascular Disease (CVD)
Year2017
CountryCanada|Denmark|Germany|Netherlands|United States
Company sponsorAkcea Therapeutics
Other ID(s)ISIS 681257-CS6|2016-003373-18
Vector information
VectorGalNAc

Clinical Result

Cohort1: ISIS 681257_dose level1_Q4W
Administration route subcutaneous injection
Dosage ISIS 681257, 20 mg, once Q4W, for up to 49 weeks and a maximum of 13 doses
Pts 48
Age Adult, Older_Adult
Adverse reactions 0/48(All-cause mortality); 7/48(Cardiac disorders; Gastrointestinal disorders; General disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders)
References : PMID：31893580
Cohort2: ISIS 681257_dose level2_Q4W
Administration route subcutaneous injection
Dosage ISIS 681257, 40 mg, once Q4W, for up to 49 weeks and a maximum of 13 doses
Pts 48
Age Adult, Older_Adult
Adverse reactions 0/48(All-cause mortality); 7/48(Cardiac disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Musculoskeletal and connective tissue disorders; Psychiatric disorders; Reproductive system and breast disorders)
References : PMID：31893580
Cohort3: ISIS 681257_dose level3_Q4W
Administration route subcutaneous injection
Dosage ISIS 681257, 60 mg, once Q4W, for up to 49 weeks and a maximum of 13 doses
Pts 17
Age Adult, Older_Adult
Adverse reactions 1/47(All-cause mortality); 7/47(Cardiac disorders; Injury, poisoning and procedural complications; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Vascular disorders)
References : PMID：31893580
Cohort4: ISIS 681257_dose level1_Q2W
Administration route subcutaneous injection
Dosage ISIS 681257, 20 mg, once Q2W, for up to 51 weeks and a maximum of 26 doses
Pts 48
Age Adult, Older_Adult
Adverse reactions 0/48(All-cause mortality); 3/48(Cardiac disorders; Gastrointestinal disorders; Metabolism and nutrition disorders)
References : PMID：31893580
Cohort5: ISIS 681257_dose level1_QW
Administration route subcutaneous injection
Dosage ISIS 681257, 20 mg, once QW, for up to 52 weeks and a maximum of 52 doses
Pts 48
Age Adult, Older_Adult
Adverse reactions 1/48(All-cause mortality); 4/48(Infections and infestations; Injury, poisoning and procedural complications; Nervous system disorders; Psychiatric disorders; Surgical and medical procedures)
References : PMID：31893580
Cohort6: Placebo
Administration route subcutaneous injection
Pts 47
Age Adult, Older_Adult
Adverse reactions 0/47(All-cause mortality); 3/47(Cardiac disorders; General disorders; Injury, poisoning and procedural complications; Nervous system disorders)
References : PMID：31893580

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