Primary-outcome results across pivotal trials
Per-arm reported values from Phase 2/3 and Phase 3 trials with results posted to ClinicalTrials.gov.
| Trial | Indication | Primary endpoint | Arm | Value |
|---|---|---|---|---|
| NCT01507831 | Hypercholesterolemia | Percentage of Participants Who Experienced Adverse Events (AEs) Up to 10 weeks after last study drug administration (maximum of 86 weeks) | Alirocumab 150 mg Q2W | 81.0 percentage of participants |
| Alirocumab 150 mg Q2W | 0.5 percentage of participants | |||
| Alirocumab 150 mg Q2W | 18.7 percentage of participants | |||
| Alirocumab 150 mg Q2W | 7.2 percentage of participants | |||
| Placebo Q2W | 1.3 percentage of participants | |||
| Placebo Q2W | 19.5 percentage of participants | |||
| Placebo Q2W | 82.5 percentage of participants | |||
| Placebo Q2W | 5.8 percentage of participants | |||
| NCT01617655 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - ITT Analysis From Baseline to Week 52 | Alirocumab 150 mg Q2W | -45.7 percent change (±3.5 Standard Error) |
| Placebo Q2W | -6.6 percent change (±4.9 Standard Error) | |||
| NCT01623115 ODYSSEY FH I | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis From Baseline to Week 52 | Alirocumab 75 mg/Up to 150 mg Q2W | -48.8 percent change (±1.6 Standard Error) |
| Placebo | 9.1 percent change (±2.2 Standard Error) | |||
| NCT01644175 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis From Baseline to Week 52 | Alirocumab 75/150 mg Q2W | -48.2 percent change (±1.9 Standard Error) |
| Placebo Q2W | -2.3 percent change (±2.7 Standard Error) | |||
| NCT01644188 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-treat (ITT) Analysis From Baseline to Week 52 | Alirocumab 75 /up to 150 mg Q2W | -50.6 percent change (±1.4 Standard Error) |
| Ezetimibe 10 mg | -20.7 percent change (±1.9 Standard Error) | |||
| NCT01644474 ODYSSEY MONO | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis From Baseline to Week 24 | Alirocumab 75/Up to 150 mg Q2W | -47.2 percent change (±3.0 Standard Error) |
| Ezetimibe 10 mg | -15.6 percent change (±3.1 Standard Error) | |||
| NCT01663402 | Atherosclerosis | Time to First Occurrence of Major Adverse Cardiovascular Event (MACE); Percentage of Observed Participants With Outcome Measure Events During the Study From randomization up to 64 months | Alirocumab 75 mg Q2W/Up to 150 mg Q2W | 9.5 percentage of participants |
| Placebo | 11.1 percentage of participants | |||
| NCT01709500 ODYSSEY FH II | — | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent--to--Treat (ITT) Analysis From Baseline to Week 52 | Alirocumab 75 mg/up to 150 mg | -48.7 percent change (±1.9 Standard Error) |
| Placebo | 2.8 percent change (±2.8 Standard Error) | |||
| NCT01709513 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent--To-Treat (ITT) Analysis From Baseline to Week 24 | Alirocumab 75 mg/ up to 150 mg | -45.0 percent change (±2.2 Standard Error) |
| Ezetimibe | -14.6 percent change (±2.2 Standard Error) | |||
| NCT01730040 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-treat (ITT) Analysis From Baseline to Week 24 | Alirocumab 75 mg/up to 150 mg + Atorvastatin 20 mg | -44.1 percent change (±4.5 Standard Error) |
| Alirocumab 75 mg/up to 150 mg + Atorvastatin 40 mg | -54 percent change (±4.3 Standard Error) | |||
| Atorvastatin 40 mg | -5 percent change (±4.6 Standard Error) | |||
| Atorvastatin 80 mg | -4.8 percent change (±4.2 Standard Error) | |||
| Ezetimibe 10 mg + Atorvastatin 20 mg | -20.5 percent change (±4.7 Standard Error) | |||
| Ezetimibe 10 mg + Atorvastatin 40 mg | -22.6 percent change (±4.3 Standard Error) | |||
| Rosuvastatin 40 mg | -21.4 percent change (±4.2 Standard Error) | |||
| NCT01730053 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis From Baseline to Week 24 | Alirocumab 75 mg/up to 150 mg + Rosuvastatin 10 mg | -50.6 percent change (±4.2 Standard Error) |
| Alirocumab 75 mg/up to 150 mg + Rosuvastatin 20 mg | -36.3 percent change (±7.1 Standard Error) | |||
| Ezetimibe 10 mg + Rosuvastatin 10 mg | -14.4 percent change (±4.4 Standard Error) | |||
| Ezetimibe 10 mg + Rosuvastatin 20 mg | -11.0 percent change (±7.2 Standard Error) | |||
| Rosuvastatin 20 mg | -16.3 percent change (±4.1 Standard Error) | |||
| Rosuvastatin 40 mg | -15.9 percent change (±7.1 Standard Error) | |||
| NCT01926782 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C in Participants Not Receiving Concomitant Statin Therapy - ITT Analysis From Baseline to Week 24 | Alirocumab 300 mg Q4W/Up 150 mg Q2W Without Concomitant Statin | -56.9 percent change (±1.8 Standard Error) |
| Alirocumab 300 mg Q4W/Up 150 mg Q2W Without Concomitant Statin | -52.7 percent change (±1.9 Standard Error) | |||
| Alirocumab 75 mg Q2W/Up 150 mg Q2W Without Concomitant Statin | -54 percent change (±3.6 Standard Error) | |||
| Alirocumab 75 mg Q2W/Up 150 mg Q2W Without Concomitant Statin | -50.2 percent change (±3.7 Standard Error) | |||
| Placebo Q2W Without Concomitant Statin | -0.3 percent change (±2.7 Standard Error) | |||
| Placebo Q2W Without Concomitant Statin | -1.6 percent change (±2.6 Standard Error) | |||
| NCT01926782 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C in Participants Receiving Concomitant Statin Therapy - Intent-to-Treat (ITT Analysis) From Baseline to Week 24 | Alirocumab 300 mg Q4W/Up 150 mg Q2W With Concomitant Statin | -65.8 percent change (±1.4 Standard Error) |
| Alirocumab 300 mg Q4W/Up 150 mg Q2W With Concomitant Statin | -58.8 percent change (±1.6 Standard Error) | |||
| Alirocumab 75 mg Q2W/Up 150 mg Q2W With Concomitant Statin | -57.9 percent change (±2.8 Standard Error) | |||
| Alirocumab 75 mg Q2W/Up 150 mg Q2W With Concomitant Statin | -51.6 percent change (±3.3 Standard Error) | |||
| Placebo Q2W With Concomitant Statin | -0.1 percent change (±2.3 Standard Error) | |||
| Placebo Q2W With Concomitant Statin | 0.8 percent change (±2.0 Standard Error) | |||
| NCT01954394 ODYSSEY OLE | Hypercholesterolemia | Percentage of Participants Who Experienced Adverse Events (AEs) Up to 10 weeks after last study drug administration (maximum of 176 weeks) | Alirocumab to Alirocumab 75 or 150 mg Q2W | 3.5 percentage of participants |
| Alirocumab to Alirocumab 75 or 150 mg Q2W | 87.3 percentage of participants | |||
| Alirocumab to Alirocumab 75 or 150 mg Q2W | 22.0 percentage of participants | |||
| Alirocumab: All Participants | 21.5 percentage of participants | |||
| Alirocumab: All Participants | 86.2 percentage of participants | |||
| Alirocumab: All Participants | 3.4 percentage of participants | |||
| Placebo to Alirocumab 75 or 150 mg Q2W | 3.0 percentage of participants | |||
| Placebo to Alirocumab 75 or 150 mg Q2W | 83.9 percentage of participants | |||
| Placebo to Alirocumab 75 or 150 mg Q2W | 20.6 percentage of participants | |||
| NCT02023879 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT Analysis) From Baseline to Week 24 | Alirocumab 150 mg Q4W/Up to 150 mg Q2W | -51.7 percent change (±2.3 Standard Error) |
| Alirocumab 75 mg Q2W/Up to 150 mg Q2W (Calibrator) | -53.5 percent change (±1.6 Standard Error) | |||
| Placebo Q2W | 4.7 percent change (±2.3 Standard Error) | |||
| NCT02107898 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT Analysis) From Baseline to Week 24 | Alirocumab 75 mg/Up to 150 mg Q2W | -62.5 percent change (±1.3 Standard Error) |
| Placebo Q2W | 1.6 percent change (±1.8 Standard Error) | |||
| NCT02289963 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-Treat (ITT) Analysis From Baseline to Week 24 | Alirocumab 75 mg Q2W/Up to 150 mg Q2W | -57.1 percent change (±3.0 Standard Error) |
| Placebo Q2W | 6.3 percent change (±2.9 Standard Error) | |||
| NCT02326220 ODYSSEY ESCAPE | — | Change in Standardized Rate of Apheresis Treatments From Week 7 to Week 18 Week 7 to Week 18 (before start of open-label treatment) | Alirocumab 150 mg Q2W (Double Blind Period) | 0.128 Treatments (±0.242 Standard Deviation) |
| Placebo Q2W (Double Blind Period) | 0.806 Treatments (±0.191 Standard Deviation) | |||
| NCT02476006 | Hypercholesterolemia | Percentage of Participants With Treatment Emergent Adverse Events (TEAEs) From first injection of investigational medicinal product (IMP) up to 2 weeks after last dose of study drug (Week 120) | Alirocumab | 16.2 percentage of participants |
| Alirocumab | 0.2 percentage of participants | |||
| Alirocumab | 4.5 percentage of participants | |||
| Alirocumab | 71.6 percentage of participants | |||
| NCT02584504 ODYSSEY-NIPPON | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 12- Intent to Treat (ITT) Analysis From Baseline to Week 12 | Alirocumab 150 mg Q2W | -70.1 percent change (±2.3 Standard Error) |
| Alirocumab 150 mg Q4W | -43.8 percent change (±2.2 Standard Error) | |||
| Placebo Q2W | -4.3 percent change (±2.2 Standard Error) | |||
| NCT02585778 | Hypercholesterolemia | Percent Change From Baseline in Calculated LDL-C at Week 24 - Intent-to-treat (ITT) Analysis From Baseline to Week 24 | Alirocumab 75 mg Q2W/Up to 150 mg Q2W: T1DM Participants | -51.8 percent change (±3.7 Standard Error) |
| Alirocumab 75 mg Q2W/Up to 150 mg Q2W: T2DM Participants | -48.2 percent change (±1.6 Standard Error) | |||
| Placebo Q2W: T1DM Participants | -3.9 percent change (±5.3 Standard Error) | |||
| Placebo Q2W: T2DM Participants | 0.8 percent change (±2.2 Standard Error) | |||
| NCT02585778 | Hypercholesterolemia | Percentage of Participants Who Experienced Treatment-Emergent Adverse Events (AEs) From Baseline up to 10 weeks after last study drug administration (maximum of 32 weeks) | Alirocumab 75 mg Q2W/Up to 150 mg Q2W | 0 percentage of participants |
| Alirocumab 75 mg Q2W/Up to 150 mg Q2W | 64.5 percentage of participants | |||
| Alirocumab 75 mg Q2W/Up to 150 mg Q2W | 9.0 percentage of participants | |||
| Alirocumab 75 mg Q2W/Up to 150 mg Q2W | 4.9 percentage of participants | |||
| Placebo Q2W | 2.4 percentage of participants | |||
| Placebo Q2W | 64.1 percentage of participants | |||
| Placebo Q2W | 9.4 percentage of participants | |||
| Placebo Q2W | 0.6 percentage of participants | |||
| NCT02715726 ODYSSEY EAST | Hypercholesterolemia | Percent Change From Baseline in Calculated Low Density Lipoprotein Cholesterol at Week 24: Intent-to-treat (ITT) Analysis From Baseline to Week 24 | Alirocumab 75 mg Q2W/up to 150 mg Q2W | -56.0 percent change (±1.5 Standard Error) |
| Ezetimibe 10 mg | -20.3 percent change (±2.0 Standard Error) | |||
| NCT03156621 ODYSSEY HoFH | Homozygous Familial Hypercholesterolemia | Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 12 (Intent-to-Treat [ITT] Estimand) Baseline to Week 12 | Alirocumab 150 mg SC Q2W | -26.9 Percentage of change (±4.6 Standard Error) |
| Placebo in DBTP | 8.6 Percentage of change (±6.3 Standard Error) | |||
| NCT03415178 | Hypercholesterolemia | Percentage of SYDNEY-Associated Product Technical Complaints (PTCs) (by Type) at the Unsupervised Injections: Single-Arm Period From Week 4 up to Week 12 | New Auto-Injector Device (SYDNEY) | 0.5 percentage of PTCs |
| New Auto-Injector Device (SYDNEY) | 0 percentage of PTCs | |||
| New Auto-Injector Device (SYDNEY) | 0 percentage of PTCs | |||
| NCT03415178 | Hypercholesterolemia | Percentage of SYDNEY-Associated Product Technical Complaints (PTCs) (Overall) at the Unsupervised Injections: Single-Arm Period From Week 4 up to Week 12 | New Auto-Injector Device (SYDNEY) | 0.5 percentage of PTCs |
| NCT03510715 | Hypercholesterolemia | Percent Change From Baseline in Low-Density Lipoprotein Cholesterol (LDL-C) at Week 12: Intent-to-Treat (ITT) Analysis Baseline to Week 12 | Alirocumab | -4.1 percent change (±9.0 Standard Error) |
| NCT03510884 | Hypercholesterolemia | DB Period: Percent Change From Baseline in Low Density Lipoprotein Cholesterol (LDL-C) at Week 24: Intent-to-treat (ITT) Estimand Baseline, Week 24 | DB Period: Alirocumab Q2W | -33.6 percent change (±3.4 Standard Error) |
| DB Period: Alirocumab Q4W | -38.2 percent change (±4.0 Standard Error) | |||
| DB Period: Placebo Q2W | 9.7 percent change (±4.3 Standard Error) | |||
| DB Period: Placebo Q4W | -4.4 percent change (±3.7 Standard Error) |