| Administration route |
subcutaneous injection |
| Dosage |
Mipomersen Sodium, 200 mg (for participants weighed ≥ 50 kg)/160 mg (for participants weighed <50 kg), once a week for up to 4 years |
| Pts |
144 |
| Age |
Child, Adult, Older_Adult |
| Outcome |
Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C):week 26 (n = 130):-28.5(-31.9 to -25.1); week 52 (n = 111):-27(-31.2 to -22.8); week 76 (n = 66):-27.3(-33 to -21.6); week 104 (n = 57):-27.9(-33.9 to -21.8); week 130 (n = 42):-21.9(-31.1 to -12.7); week 156 (n = 30):-21.4(-31.2 to -11.7); week 182 (n = 26):-23.6(-36.6 to -10.6); week 208 (n = 27):-26.3(-36.4 to -16.2); week 234 (n = 17):-22.5(-34.3 to -10.6); 24 weeks post last dose (n=117):1.6(-2.6 to 5.9)|Percent Change From Baseline in Apolipoprotein B (Apo B):week 26 (n = 130):-28.9(-32 to -25.8); week 52 (n = 111):-28.1(-32 to -24.2); week 76 (n = 66):-30.3(-34.7 to -26); week 104 (n = 57):-31.2(-36.5 to -25.9); week 130 (n = 43):-29.1(-35.7 to -22.5); week 156 (n = 30):-30.2(-38.1 to -22.2); week 182 (n = 26):-31.1(-39.9 to -22.2); week 208 (n = 27):-33.3(-40.8 to -25.9); week 234 (n = 17):-31.4(-38.7 to -24.1); 24 weeks post last dose (n=117):-3.46(-6.9 to 0)|Percent Change From Baseline in Total Cholesterol:week 26 (n = 130):-21.7(-24.4 to -18.9); week 52 (n = 111):-20.4(-23.9 to -16.8); week 76 (n = 66):-20.1(-24.6 to -15.5); week 104 (n = 57):-19.8(-24.8 to -14.7); week 130 (n = 43):-14.9(-22.1 to -7.8); week 156 (n = 30):-14.4(-22.3 to -6.6); week 182 (n = 26):-14.3(-25 to -3.5); week 208 (n = 27):-16.5(-24.2 to -8.8); week 234 (n = 17):-12.5(-21.5 to -3.4); 24 weeks post last dose (n=117):1.94(-1.5 to 5.4)|Percent Change From Baseline in Non High Density Lipoprotein Cholesterol (Non-HDL-C):week 26 (n = 130):-27.2(-30.4 to -24.1); week 52 (n = 111):-25.4(-29.5 to -21.3); week 76 (n = 66):-25(-30.4 to -19.7); week 104 (n = 57):-26.2(-32 to -20.4); week 130 (n = 43):-20.7(-29.1 to -12.3); week 156 (n = 30):-20(-29.6 to -10.3); week 182 (n = 26):-21.7(-34.7 to -8.7); week 208 (n = 27):-23.9(-33.7 to -14.1); week 234 (n = 17):-19.9(-31.5 to -8.2); 24 weeks post last dose (n=117):2.5(-1.8 to 6.7) |
| Adverse reactions |
36/141(Blood and lymphatic system disorders; Cardiac disorders; Gastrointestinal disorders; General disorders; Hepatobiliary disorders; Immune system disorders; Infections and infestations; Injury, poisoning and procedural complications; Metabolism and nutrition disorders; Musculoskeletal and connective tissue disorders; Neoplasms benign, malignant and unspecified(incl cysts and polyps); Nervous system disorders; Psychiatric disorders; Renal and urinary disorders; Respiratory, thoracic and mediastinal disorders; Surgical and medical procedures; Vascular disorders) |
| References |
PMID:
24366918
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