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 GTC0051
Trial ID NCT00607373
Disease Dyslipidemias | Familial Hypercholesterolemia | Lipid Metabolism Disorder | Hyperlipoproteinemia Type II | Genetic Disease | Metabolic Disease
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 the Safety and Efficacy of Mipomersen as Add-on Therapy in Homozygous Familial Hypercholesterolemia Subjects
Year2007
CountryBrazil|Canada|Singapore|South Africa|China|United Kingdom|United States
Company sponsorKastle Therapeutics, LLC
Other ID(s)301012CS5|2005-003449-15
Vector information
VectorNo vector was used

Clinical Result

Cohort1: mipomersen
Administration route subcutaneous injection
Dosage mipomersen, 200 mg, 1 mL, once a week for 26 weeks; mipomersen, 160 mg, 0.8 mL, once a week for 26 weeks (Subjects weighing less than 50 kg)
Pts 34
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point:Mean (Standard Deviation):-3.31(17.06)|LDL-C at Baseline and the Primary Efficacy Time Point (PET):Baseline Mean (Standard Deviation):438.9(138.6); PET:326.2(121.3)
Adverse reactions 2/34(Cardiac disorders; Injury, poisoning and procedural complications)
References PMID: 20227758
Cohort2: Placebo
Administration route subcutaneous injection
Dosage Placebo, 1 mL, once a week for 26 weeks; Placebo, 0.8 mL, once a week for 26 weeks (Subjects weighing less than 50 kg)
Pts 17
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C) at Primary Efficacy Time Point:Mean (Standard Deviation):-3.31(17.06)|LDL-C at Baseline and the Primary Efficacy Time Point (PET):Baseline Mean (Standard Deviation):400.2(141.5); PET:388.2(150.5)
Adverse reactions 1/17(Renal and urinary disorders)
References PMID: 20227758

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