Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT01709500 | Study of Alirocumab (REGN727/SAR236553) in Patients With heFH (Heterozygous Familial Hypercholesterolemia) Who Are Not Adequately Controlled With Their LMT (Lipid-Modifying Therapy) | PHASE3 | COMPLETED | 249 | — | — | Dec 1, 2012 | Jan 1, 2015 | Oct 28, 2015 | 24 | Czechia, Netherlands +2 |
Calculated LDL-C values were obtained using the Friedewald formula. Adjusted Least- squares (LS) means and standard errors at Week 24 were obtained from a mixed -effect model with repeated measures (MMRM) to account for missing data. All available post -baseline data from Week 4 to Week 52 regardless of status on- or off-treatment were used in the model.
| Arm | Type | Description |
|---|---|---|
| Alirocumab 75 mg/up to 150 mg | EXPERIMENTAL | Alirocumab 75 mg every two weeks (Q2W) added to stable dose of statin with or without LMT for 78 weeks. Alirocumab dose up-titrated to 150 mg Q2W from Week 12 when LDL-C levels ≥70 mg/dL at Week 8. |
| Placebo | PLACEBO_COMPARATOR | Placebo matched to alirocumab SC injection for 78-week treatment duration. |
| Name | Type | Description |
|---|---|---|
| LMT (atorvastatin, simvastatin, or rosuvastatin) | DRUG | - |
| alirocumab | DRUG | Alirocumab administered as a subcutaneous (SC) injection of 1 mL into the abdomen, thigh, or outer area of the upper arm. |
| Placebo | DRUG | Placebo matched to alirocumab administered as a SC injection of 1 mL into the abdomen, thigh, or outer area of the upper arm. |
Inclusion Criteria: 1. Patients with heFH\* who are not adequately controlled\*\* with a maximally-tolerated daily dose\*\*\* of statin with or without other LMT, at a stable dose prior to the screening visit (week -2). \*Diagnosis of heFH must be made either by genotyping or by clinical criter...