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 GTC0066
Trial ID NCT00770146
Disease Coronary Artery Disease | Familial Hypercholesterolemia
Altered gene APOB
Therapeutic/Target gene Target gene
TherapyASO
Treatment ISIS 301012|mipomersen sodium|Kynamro
Co-treatment riboflavin
Location approved US, Mexico, Argentina, South Korea
PhasePhase3
Recruitment statusCompleted
TitleA Randomized, Double-Blind, Placebo-Controlled Study to Assess the Safety and Efficacy of ISIS 301012 (Mipomersen) as Add-on Therapy in High Risk Hypercholesterolemic Patients
Year2008
CountryUnited States
Company sponsorKastle Therapeutics, LLC
Other ID(s)301012-CS12
Vector information
VectorNo vector was used

Clinical Result

Cohort1: Placebo
Administration route subcutaneous injection
Dosage Placebo, 1 mL (9 mg of sodium chloride, 0.004 mg of riboflavin), once a week for 26 weeks
Pts 52
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point:Median (Inter-Quartile Range):-3.4(-14.8 to 11.8)|Low-density Lipoprotein Cholesterol (LDL-C) at Baseline and at the Primary Efficacy Time Point:Baseline Median (Inter-Quartile Range):112(101 to 134); Primary efficacy time point:109(90 to 128)
Adverse reactions 4/52(Cardiac disorders; General disorders; Respiratory, thoracic and mediastinal disorders)
References PMID: 24013058
Cohort2: mipomersen
Administration route subcutaneous injection
Dosage mipomersen, 200 mg, once a week for 26 weeks
Pts 105
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point:Median (Inter-Quartile Range):-40.2(-56.4 to -19.1)|Low-density Lipoprotein Cholesterol (LDL-C) at Baseline and at the Primary Efficacy Time Point:Baseline Median (Inter-Quartile Range):116(106 to 137); Primary efficacy time point:69(51 to 98)
Adverse reactions 7/105(Cardiac disorders; General disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Vascular disorders)
References PMID: 24013058

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