Phase 2
N=77
Study of the Safety and Efficacy of REGN727/SAR236553 in Patients With HeFH Hypercholesterolemia
Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT01266876 ↗Enrolled (actual)
77
Serious AEs
1.3%
Results posted
Sep 2015
Primary outcome: Primary: Percent Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis — -10.7; -28.9; -31.5; -42.5 percent change — p=0.0000
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- Alirocumab (Drug); Placebo (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Regeneron Pharmaceuticals
- Primary completion
- Nov 2011
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percent Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis |
-10.7; -28.9; -31.5; -42.5; -67.9 | 0.0000 sig |
| SECONDARY Absolute Change From Baseline in Calculated LDL-C at Week 12 - On-treatment Analysis |
-19.1; -42.2; -51.3; -66.9; -102.5 | — |
| SECONDARY Percentage of Participants Achieving Calculated LDL-C <100 mg/dL (2.59 mmol/L) at Week 12 - On-treatment Analysis |
13.3; 26.7; 18.8; 66.7; 93.8 | — |
| SECONDARY Percentage of Participants Achieving LDL-C < 70 mg/dL (1.81 mmol/L) at Week 12 - On-treatment Analysis |
0.0; 13.3; 12.5; 46.7; 81.3 | — |
| SECONDARY Percent Change From Baseline in Total Cholesterol at Week 12 - On-treatment Analysis |
-8.5; -18.1; -19.7; -25.7; -43.6 | — |
| SECONDARY Absolute Change From Baseline in Total Cholesterol at Week 12 - On-treatment Analysis |
-20.3; -40.3; -50.1; -61.7; -99.9 | — |
| SECONDARY Percent Change From Baseline in High Density Lipoprotein Cholesterol (HDL-C) at Week 12 - On-treatment Analysis |
2.2; 7.9; 6.5; 1.00; 12.3 | — |
| SECONDARY Absolute Change From Baseline in HDL-C at Week 12 - On-treatment Analysis |
0.4; 4.2; 3.3; 4.5; 6.0 | — |
| SECONDARY Percent Change From Baseline in Triglycerides at Week 12 - On-treatment Analysis |
-10.6; -16.7; -13.2; -4.9; -16.2 | — |
| SECONDARY Absolute Change From Baseline in Triglycerides at Week at 12 - On-treatment Analysis |
-11.0; -20.0; -13.5; -3.5; -14.8 | — |
| SECONDARY Percent Change From Baseline in Non-HDL-C at Week 12 - On-treatment Analysis |
-11.3; -26.8; -27.4; -39.0; -57.9 | — |
| SECONDARY Absolute Change From Baseline in Non-HDL-C at Week 12 - On-treatment Analysis |
-22.8; -46.0; -52.3; -70.6; -103.6 | — |
| SECONDARY Percent Change From Baseline in Apo Lipoprotein B (Apo-B) at Week 12 - On-treatment Analysis |
-6.4; -20.9; -20.9; -28.4; -50.2 | — |
| SECONDARY Absolute Change From Baseline in Apo-B at Week 12 - On-treatment Analysis |
-9.3; -24.3; -28.7; -34.7; -64.4 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein - A1 (Apo-A1) at Week 12 - On-treatment Analysis |
-5.3; 2.4; 1.7; 5.6; 8.8 | — |
| SECONDARY Absolute Change From Baseline in Apo-A1 at Week 12 - On-treatment Analysis |
-9.7; 3.4; 2.8; 7.8; 11.0 | — |
| SECONDARY Absolute Change in the Ratio ApoB/ApoA-1 From Baseline to Week 12 - On-treatment Analysis |
-0.03; -0.18; -0.20; -0.25; -0.49 | — |
| SECONDARY Percent Change From Baseline in Lipoprotein(a) at Week 12 - On-treatment Analysis |
-3.9; -10.1; -7.5; -15.3; -23.4 | — |
| SECONDARY Absolute Change From Baseline in Lipoprotein(a) at Week 12 - On-treatment Analysis |
-2.0; -1.5; -1.8; -8.0; -11.5 | — |
Summary
The purpose of this study is to assess the efficacy and safety of REGN727/SAR236553 in participants diagnosed with heterozygous familial hypercholesterolemia (heFH)
Eligibility Criteria
Inclusion Criteria
- Must meet the World Health Organization criteria for heFH
- Participants must be on a stable statin dose, with or without ezetimibe, for at least 6 weeks before screening
- Serum LDL-C levels ≥ 100 mg/dL at screening
- Willing to follow the NCEP ATPIII TLC diet, or an equivalent diet plan, starting at screening and continuing until the last study visit
- A negative urine/serum pregnancy test at each screening visit and start of the study, for women of childbearing potential
Key Exclusion Criteria
- Participants with homozygous FH (clinically or by previous genotyping)
- Use of a medication (other than a statin or EZE) to alter serum lipids within 42 days (6 weeks) before screening including, but not limited to:
- Fibrates
- Niacin (>500 mg/day)
- Omega-3 fatty acids (>1000 mg/day of DHA/EPA)
- Bile acid resins
- Use of nutraceuticals or OTC medications that may alter lipid levels that are not stable for at least 6 weeks before screening and are not planned to remain constant throughout the study. Examples include:
- Omega-3 fatty acids (≤1000 mg/day of DHA/EPA)
- Niacin (≤500 mg/day)
- Plant stanols, such as found in Benecol, flax seed oil, psyllium
- Red yeast rice
- Disorders known to influence lipid levels, such as nephrotic syndrome, significant liver disease, Cushing's disease, untreated hypothyroidism (patients on stable thyroid replacement for at least 12 weeks before the full screening visit, who are metabolically euthyroid by thyroid-stimulating hormone (TSH) testing are allowed)
- Use of thyroid medications (except for replacement therapy which has been stable for at least 12 weeks before the full screening visit)
- Fasting serum TG >350 mg/dL screening
- LDL apheresis within 12 months before screening
Data sourced from ClinicalTrials.gov (NCT01266876). 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.