Phase 2
Completed N=58
Open-Label Extension of Study R727-CL-1003 (NCT01266876) to Evaluate the Long-Term Safety and Efficacy of Alirocumab (REGN727) in Participants With Heterozygous Familial Hypercholesterolemia (HeFH)
Hypercholesterolemia · Heterozygous Familial Hypercholesterolemia
Source: ClinicalTrials.gov NCT01576484 ↗
Enrolled (actual)
58
Serious AEs
20.7%
Results posted
Aug 2020
Primary outcomePrimary: Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death — 12; 42; 54; 4 Participants
Summary
The primary objective of the study was to assess the long-term safety and tolerability of alirocumab in patients with heFH who were receiving concomitant treatment with hydroxymethyl glutaryl-coenzyme A (HMG-CoA) reductase inhibitors (statins), with or without other lipid-modifying therapies (LMTs).
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants Who Experienced Treatment Emergent Adverse Events (TEAEs), Serious TEAEs and TEAEs Leading to Death |
12; 42; 54; 4; 8; 12 | — |
| SECONDARY Percent Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline in the Current Study to Week 24 |
-73.15; -63.40; -65.35 | — |
| SECONDARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in Current Study to Week 12 |
-55.93; -63.94; -62.22 | — |
| SECONDARY Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), and Total Cholesterol From Baseline in Current Study to Week 24 |
-52.23; -50.52; -50.86; -56.75; -55.43; -55.71 | — |
| SECONDARY Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), and Total Cholesterol From Baseline in Current Study to Week 12 |
-40.69; -48.78; -47.16; -47.81; -54.97; -53.44 | — |
| SECONDARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in Current Study to Week 52 |
-54.57; -55.67; -55.43 | — |
| SECONDARY Percentage of Participants With Low Density Lipoprotein Cholesterol (LDL-C) Less Than (<) 70 Milligrams Per Deciliter (mg/dL) for Prior Myocardial Infarction (MI)/Stroke, or <100 mg/dL [2.59 mmol/L] for Participants Without Prior MI/Stroke at Week 24 |
100.00; 90.91; 92.73 | — |
| SECONDARY Percent Change in Lipoprotein a (Lp[a]) at Week 24 |
-27.91; -29.41; -29.11 | — |
| SECONDARY Percent Change in High Density Lipoprotein Cholesterol (HDL-C) at Week 24 and Week 12 |
2.61; 7.14; 6.17; 1.46; 10.43; 8.51 | — |
| SECONDARY Percent Change in Lipoprotein a at Week 12 |
-20.30; -26.98; -25.64 | — |
| SECONDARY Percent Change in Triglycerides (TG) at Week 24 and Week 12 |
0.19; -2.01; -1.54; -6.40; 0.52; -0.96 | — |
| SECONDARY Percent Change in Apolipoprotein A-1 (Apo A-1) at Week 24 and Week 12 |
5.41; 4.78; 4.91; 11.10; 8.64; 9.13 | — |
| SECONDARY Percent Change in Low Density Lipoprotein Cholesterol (LDL-C) From Baseline in the Current Study to the End of Treatment |
-52.20; -58.46; -57.36 | — |
| SECONDARY Percentage of Participants With Low Density Lipoprotein Cholesterol (LDL-C) Less Than (<) 70 Milligrams Per Deciliter (mg/dL) for Prior MI/Stroke, or <100 mg/dL [2.59 mmol/L] for Participants Without Prior MI/Stroke at Week 12, 52 and End of Treatment |
83.33; 90.91; 89.29; 75.00; 83.33; 81.48 | — |
| SECONDARY Absolute Change in Low-density Lipoprotein Cholesterol (LDL-C) From Baseline in the Current Study to Weeks 12, 24, 52, and End of Treatment |
-92.0; -96.8; -95.8; -113.2; -95.3; -98.9 | — |
| SECONDARY Percent Change in Apolipoprotein (Apo) B, Non-High Density Lipoprotein Cholesterol (HDL-C), Total Cholesterol, Lipoprotein a (Lp[a]), HDL-C, Triglycerides, and Apolipoprotein A-1 From Baseline in Current Study to Week 52 and End of Treatment |
-43.04; -45.17; -44.73; -45.02; -48.07; -47.53 | — |
| SECONDARY Change in Ratio in Apolipoprotein (Apo) B/Apo A-1 From Baseline in Current Study to Week 12, 24, 52, and End of Treatment. |
-0.397; -0.456; -0.444; -0.450; -0.460; -0.458 | — |
| SECONDARY Percentage of Participants With Apolipoprotein (Apo) B <80 mg/dL at Week 12, 24, 52, and End of Treatment |
66.67; 79.55; 76.79; 91.67; 81.82; 83.93 | — |
| SECONDARY Percentage of Participants With Non-High Density Lipoprotein Cholesterol (HDL-C) <100 mg/dL (2.59 mmol/L) at Week 12, 24, 52, and End of Treatment |
58.33; 75.00; 71.43; 91.67; 81.82; 83.93 | — |
| SECONDARY Percentage of Participants With Low Density Lipoprotein Cholesterol (LDL-C) Less Than (<) 70 Milligrams Per Deciliter (mg/dL) and/or ≥ 50% Reduction in LDL-C (if LDL-C >70 mg/dL [1.81 mmol/L]) at Week 12, 24, 52, and End of Treatment |
66.67; 86.36; 82.14; 100.00; 81.82; 85.45 | — |
Eligibility Criteria
Key Inclusion Criteria
- Prior participation in and the successful completion of the R727-CL-1003 study (NCT01266876).
- Patients must be on a stable daily statin regimen for at least 3 weeks before prior to entry into the study
- A negative urine pregnancy at the screening/baseline visit for women of childbearing potential
Key Exclusion Criteria
- Reported a drug-related serious adverse event (SAE) or drug-related clinical or laboratory adverse event (AE) in the R727-CL-1003 study that resulted in early termination or withdrawal
- Significant protocol deviation in R727-CL-1003, such as non-compliance by the investigator or patient
- Low-density lipoprotein (LDL) apheresis within 12 months before the screening/baseline visit
Note: Other exclusion criteria applied
Data sourced from ClinicalTrials.gov (NCT01576484). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.