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 GTC1340
Trial ID NCT01061814
Disease Familial Hypercholesterolemia
Altered gene APOB
Therapeutic/Target gene Target gene
TherapyASO
Treatment ISIS 301012|mipomersen sodium|Kynamro
Location approved US, Mexico, Argentina, South Korea
PhasePhase1
Recruitment statusCompleted
TitleA Prospective, Randomized, Double-Blind, Placebo-Controlled, Phase 1 Study to Evaluate the Relative Bioavailability, Pharmacokinetics, Safety, and Tolerability of Daily, Thrice Weekly, and Weekly Dosing Regimens of Mipomersen Administered Subcutaneously to Healthy Volunteers
Year2010
CountryCanada
Company sponsorKastle Therapeutics, LLC
Other ID(s)MIPO3200309
Vector information
VectorNo vector was used

Clinical Result

Cohort1: mipomersen_dose level 1
Administration route subcutaneous injection
Dosage mipomersen, 30 mg QD (Day 1 to 21; 630 mg total)
Pts 21
Age Adult, Older_Adult
References PMID: 24627419
Cohort2: mipomersen_dose level 2
Administration route subcutaneous injection
Dosage mipomersen, 70 mg TIW (Day 1, 3, 5, 8, 10, 12, 15, 17, and 19; 630 mg total)
Pts 21
Age Adult, Older_Adult
References PMID: 24627419
Cohort3: mipomersen_dose level 3
Administration route subcutaneous injection
Dosage mipomersen, 200 mg QW (Day 1, 8, and 15; 600 mg total)
Pts 21
Age Adult, Older_Adult
References PMID: 24627419
Cohort4: Placebo
Administration route subcutaneous injection
Pts 21
Age Adult, Older_Adult
References PMID: 24627419

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