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 GTC0063
Trial ID NCT00707746
Disease Metabolic Disease | Lipid Metabolism Disorder | Familial Hypercholesterolemia
Altered gene APOB
Therapeutic/Target gene Target gene
TherapyASO
Treatment ISIS 301012|mipomersen sodium|Kynamro
Location approved US, Mexico, Argentina, South Korea
PhasePhase2
Recruitment statusCompleted
TitleA Randomized, Double-Blind, Placebo-Controlled Study to Assess Safety and Efficacy of ISIS 301012 Administration in High Risk Statin Intolerant Subjects
Year2008
CountryNetherlands
Company sponsorKastle Therapeutics, LLC
Other ID(s)301012-CS19|2007-005140-24
Vector information
VectorNo vector was used

Clinical Result

Cohort1: Placebo
Administration route subcutaneous injection
Dosage Placebo, 1 mL, once a week for 26 weeks
Pts 12
Age Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point:Mean (Standard Deviation):-2.0(8.40)|Low-density Lipoprotein Cholesterol at Baseline and the Primary Efficacy Time Point:Baseline Mean (Standard Deviation):243.7(65.6); Primary efficacy time point:236.3(52.8)|Summary of Participants With Adverse Events:
Adverse reactions 1/12(Cardiac disorders)
References PMID: 22507979
Cohort2: mipomersen
Administration route subcutaneous injection
Dosage mipomersen, 200 mg, weekly for 26 weeks
Pts 21
Age Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at the Primary Efficacy Time Point:Mean (Standard Deviation):-47.3(18.43)|Low-density Lipoprotein Cholesterol at Baseline and the Primary Efficacy Time Point:Baseline Mean (Standard Deviation):241.9(90.8); Primary efficacy time point:128.3(74.2)|Summary of Participants With Adverse Events:
Adverse reactions No serious clinical adverse events
References PMID: 22507979

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