Phase 3
N=62
Study of Alirocumab (REGN727/SAR236553) in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) Undergoing Low-density Lipoprotein (LDL) Apheresis Therapy
Heterozygous Familial Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT02326220 ↗Enrolled (actual)
62
Serious AEs
7.7%
Results posted
May 2020
Primary outcome: Primary: Change in Standardized Rate of Apheresis Treatments From Week 7 to Week 18 — 0.806; 0.128 Treatments — p=< 0.0001
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- Placebo (Drug); Alirocumab (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Regeneron Pharmaceuticals
- Primary completion
- Jan 2016
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Change in Standardized Rate of Apheresis Treatments From Week 7 to Week 18 |
0.806; 0.128 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline in Calculated LDL-C (Pre-apheresis) at Week 6 |
1.6; -53.7 | < 0.0001 sig |
| SECONDARY Change in Standardized Rate of Apheresis Treatments From Week 15 to Week 18 |
0.774; 0.165 | <.0001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein B (Apo B) (Pre-apheresis) to Week 6 |
1.2; -42.8 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline in Non-High-Density Lipoprotein Cholesterol (Non-HDL-C) (Pre-apheresis) to Week 6 |
2.8; -47.1 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline in Total Cholesterol (Pre-apheresis) to Week 6 |
3.1; -36.4 | < 0.0001 sig |
| SECONDARY Percent Change From Baseline in Apolipoprotein A (Apo A1) (Pre-apheresis) to Week 6 |
0; 4.2 | 0.3012 |
| SECONDARY Percentage of Participants With At Least (>=) 30% Reduction in Calculated LDL-C (Pre-apheresis) at Week 6 |
4.8; 95.1 | — |
| SECONDARY Percentage of Participants With At Least (>=) 50% Reduction in Calculated LDL-C (Pre-apheresis) at Week 6 |
0; 63.4 | — |
| SECONDARY Percent Change From Baseline in Calculated LDL-C (Pre-Apheresis) to Week 18 |
4; -42.3 | — |
| SECONDARY Percent Change From Baseline in Apolipoprotein B (Apo B) (Pre-apheresis) to Week 18 |
1.7; -33.8 | — |
| SECONDARY Percent Change From Baseline in Non-HDL-C (Pre-apheresis) to Week 18 |
4.7; -35.7 | — |
| SECONDARY Percent Change From Baseline in Total Cholesterol (Pre-apheresis) to Week 18 |
4.7; -27.1 | — |
| SECONDARY Percent Change From Baseline in Apo A1 (Pre-apheresis) to Week 18 |
-0.7; 7.8 | — |
| SECONDARY Percentage of Participants With At Least (>=) 30% Reduction in Calculated LDL-C (Pre-apheresis) at Week 18 |
0; 65.9 | — |
| SECONDARY Percentage of Participants With At Least (>=) 50% Reduction in Calculated LDL-C (Pre-apheresis) at Week 18 |
0; 43.9 | — |
| SECONDARY Change From Baseline in W-BQ22 (Well-being Questionnaire) Index Score at Week 18 |
-1.43; 0.91 | — |
| SECONDARY Percent Change From Baseline in Lipoprotein (a) (Lp [a]) (Pre-apheresis) to Week 6 |
-4; -18.1 | — |
| SECONDARY Percent Change From Baseline in High-Density Lipoprotein Cholesterol (HDL-C) (Pre-apheresis) to Week 6 |
4; 9.3 | — |
| SECONDARY Percent Change From Baseline in Triglyceride (TG) Levels (Pre-apheresis) to Week 6 |
3; -12.9 | — |
| SECONDARY Percent Change From Baseline in Lp (a) (Pre-apheresis) to Week 18 |
-1.2; -6.1 | — |
| SECONDARY Percent Change From Baseline in HDL-C (Pre-apheresis) to Week 18 |
2.4; 10.9 | — |
| SECONDARY Percent Change From Baseline in TG Levels (Pre-apheresis) to Week 18 |
4.1; -2.4 | — |
Summary
The primary objective of the study is to evaluate the effect of alirocumab 150 mg every 2 weeks (Q2W) in comparison with placebo on the frequency of low-density lipoprotein (LDL) apheresis treatments in participants with heterozygous familial hypercholesterolemia (HeFH) undergoing weekly or bi-weekly LDL apheresis therapy.
Eligibility Criteria
Inclusion Criteria
- Men and women ≥18 years of age at the time of the screening visit
- Diagnosis of HeFH (Heterozygous familial hypercholesterolemia)
- Currently undergoing LDL (low-density lipoprotein) apheresis therapy QW (weekly) or Q2W (every 2 weeks) or at least 8 weeks prior to the screening visit
Exclusion Criteria
- Homozygous FH (familial hypercholesterolemia)
- Background medical LMT (lipid-modifying therapy) (if applicable) that has not been stable for at least 8 weeks prior to the screening visit
- LDL apheresis schedule/ apheresis settings that have not been stable for at least 8 weeks prior to the screening visit
- An LDL apheresis schedule other than QW to Q2W
- Initiation of a new exercise program or exercise that has not remained stable within 8 weeks prior to the screening visit (week -2)
- Initiation of a new diet or a diet that has not been stable within 8 weeks prior to the screening visit (week -2)
- Use of nutraceuticals or over-the-counter therapies known to affect lipids, at a dose/amount that has not been stable for at least 8 weeks prior to the screening visit (week -2), or between the screening and randomization visit
- Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
- Known history of a positive test for human immunodeficiency virus
- Use of any active investigational drugs within 1 month or 5 half-lives of screening, whichever is longer
- Patients who have been treated with at least 1 dose of alirocumab or any other anti-PCSK9 monoclonal antibody in any other clinical studies
- Pregnant or breastfeeding women
Data sourced from ClinicalTrials.gov (NCT02326220). 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.