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 GTC0036
Trial ID NCT00477594
Disease Lipid Metabolism Disorder | Hyperlipoproteinemia Type II | Familial Hypercholesterolemia | Dyslipidemias
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
TitleAn Open-Label Extension Study to Assess the Long-term Safety and Efficacy of Mipomersen in Subjects With Familial Hypercholesterolemia
Year2007
CountryUnited States
Company sponsorKastle Therapeutics, LLC
Other ID(s)301012-CS17|2007-001024-12
Vector information
VectorNo vector was used

Clinical Result

Cohort1: once a week up to 3 years
Administration route subcutaneous injection
Dosage mipomersen, 200 mg, once a week up to 3 years
Pts 16
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C):Week 52 [N=13, 4]:-16.2(-41.8 to -7.8); Week 104 [N=7, 1]:-32.4(-38.8 to -16.6); Week 104/Early Termination [N=16, 5]:-13.8(-31.4 to -3.0)|Low-density Lipoprotein Cholesterol (LDL-C) Over Time:Baseline [N=16, 5]:177(134 to 205)|Week 52 [N=13, 4]:145(106 to 173)|Week 104 [N=7, 1]:121(82 to 156); Week 104/Early Termination [N=16, 5}:144(116 to 213)
Adverse reactions 4/16(Gastrointestinal disorders; General disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps))
References PMID: 20707601
Cohort2: every other week up to 3 years
Administration route subcutaneous injection
Dosage mipomersen, 200 mg, every other week up to 3 years
Pts 5
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline in Low-density Lipoprotein Cholesterol (LDL-C):Week 52 [N=13, 4]:-2.6(-10.2 to 14.8); Week 104 [N=7, 1]:30.6(30.6 to 30.6); Week 104/Early Termination [N=16, 5]:9.5(3.4 to 11.5)|Low-density Lipoprotein Cholesterol (LDL-C) Over Time:Baseline [N=16, 5]:131(124 to 147); Week 52 [N=13, 4]:127(124 to 139); Week 104 [N=7, 1]:162(162 to 162); Week 104/Early Termination [N=16, 5}:161(146 to 162)
Adverse reactions 3/5(Cardiac disorders)
References PMID: 20707601

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