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 GTC0128
Trial ID NCT01475825
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
PhasePhase3
Recruitment statusCompleted
TitleA Phase 3, Randomized, Double-Blind, Placebo-Controlled, Parallel-Group Study Followed by an Open-Label Continuation Period to Assess the Safety and Efficacy of Two Different Regimens of Mipomersen in Patients With Familial Hypercholesterolemia and Inadequately Controlled Low-Density Lipoprotein Cholesterol
Year2011
CountryArgentina|Australia|Belgium|Brazil|Canada|Croatia|Czechia|Denmark|Germany|Greece|China|Hungary|India|Israel|Italy|Korea,Republic of|Malaysia|Netherlands|New Zealand|Norway|Poland|Russian Federation|South Africa|Spain|Sweden|Turkey|Ukraine|United Kingdom|United States
Company sponsorKastle Therapeutics, LLC
Other ID(s)MIPO3801011|2011-001480-42|EFC12875
Vector information
VectorNo vector was used

Clinical Result

Cohort1: mipomersen_dose level 1
Administration route subcutaneous injection
Dosage mipomersen, 200 mg, once weekly
Pts 104
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1:Least Squares Mean (Standard Error):-27.17(5.653)
Adverse reactions 1/104(All-cause mortality); 17/104(Blood and lymphatic system disorders; Cardiac disorders; General disorders; Hepatobiliary disorders; Infections and infestations; Nervous system disorders; Renal and urinary disorders; Reproductive system and breast disorders; Respiratory, thoracic and mediastinal disorders; Vascular disorders)
References PMID: 26946290
Cohort2: Placebo_once weekly
Administration route subcutaneous injection
Dosage Placebo matching, once weekly
Pts 51
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1:Least Squares Mean (Standard Error):-6.77(6.749)
Adverse reactions 0/51(All-cause mortality); 13/51(Blood and lymphatic system disorders; Cardiac disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Pregnancy, puerperium and perinatal conditions; Renal and urinary disorders; Vascular disorders)
References PMID: 26946290
Cohort3: mipomersen_dose level 2
Administration route subcutaneous injection
Dosage mipomersen, 70 mg, thrice weekly
Pts 102
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1:-22.96(5.362)
Adverse reactions 1/102(All-cause mortality); 23/102(Cardiac disorders; Eye disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Musculoskeletal and connective tissue disorders; Nervous system disorders; Reproductive system and breast disorders; Respiratory, thoracic and mediastinal disorders; Vascular disorders)
References PMID: 26946290
Cohort4: Placebo_thrice weekly
Administration route subcutaneous injection
Dosage Placebo matching, thrice weekly
Pts 52
Age Child, Adult, Older_Adult
Outcome Percent Change From Baseline To Primary Endpoint Visit (PET) In LDL-C In Cohort 1:-10.62(5.765)
Adverse reactions 0/52(All-cause mortality); 11/52(Cardiac disorders; Gastrointestinal disorders; General disorders; Infections and infestations; Injury, poisoning and procedural complications; Nervous system disorders; Psychiatric disorders; Reproductive system and breast disorders; Respiratory, thoracic and mediastinal disorders; Vascular disorders)
References PMID: 26946290

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