Basic Information

Gene symbol APOB Synonyms FCHL2, FLDB, LDLCQ4, apoB-100, apoB-48 Type of gene protein-coding
Gene perturbation-related omics dataset PertOrg
Description apolipoprotein B

GTO ID GTC0061
Trial ID NCT00706849
Disease Familial Hypercholesterolemia | Lipid Metabolism Disorder | Hyperlipoproteinemia Type II | Dyslipidemias
Altered gene APOB
Therapeutic/Target gene Target gene
TherapyASO
Treatment ISIS 301012|mipomersen sodium|Kynamro
Location approved US, Mexico, Argentina, South Korea
PhasePhase3
Recruitment statusCompleted
TitleA Randomized, Double-Blind, Placebo-Controlled Study to Assess Efficacy and Safety of ISIS 301012 as Add-on Therapy in Heterozygous Familial Hypercholesterolemia Subjects With Coronary Artery Disease
Year2008
CountryUnited States|Canada
Company sponsorKastle Therapeutics, LLC
Other ID(s)301012-CS7
Vector information
VectorNo vector was used

Clinical Result

Cohort1: mipomersen
Administration route subcutaneous injection
Dosage mipomersen, 200 mg, once a week for 26 weeks
Pts 83
Age Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point:-28.02 (26.99)|LDL Cholesterol at Baseline and at the Primary Efficacy Time Point:Baseline:152.9(48.7); Primary efficacy time point:103.9(33.0)
Adverse reactions 6/83(Cardiac disorders; General disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Respiratory, thoracic and mediastinal disorders)
References PMID: 23060426 | 25614280
Cohort2: Placebo
Administration route subcutaneous injection
Dosage placebo, once a week for 26 weeks
Pts 41
Age Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point:5.17 (18.02)|LDL Cholesterol at Baseline and at the Primary Efficacy Time Point:Baseline:142.9(51.6); Primary efficacy time point:146.4(43.4)
Adverse reactions 2/41(Cardiac disorders)
References PMID: 23060426 | 25614280

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