Study of Alirocumab (REGN727/SAR236553) in Patients With Heterozygous Familial Hypercholesterolemia (HeFH) Undergoing Low-density Lipoprotein (LDL) Apheresis Therapy (ODYSSEY ESCAPE)

Regeneron Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Heterozygous Familial Hypercholesterolemia

Treatments: Drug: Placebo; Drug: Alirocumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT02326220

R727-CL-1216

Details and patient eligibility

About

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.

Full description

LDL apheresis removes low-density lipoproteins (LDL) that transport cholesterol in the plasma portion of the blood. This treatment is mainly used for familial hypercholesterolemia, but can be used in other rare diseases. Familial hypercholesterolemia (HeFH) is an inherited genetic condition that causes accumulation of cholesterol in the blood, which can lead to atherosclerosis and heart disease. This treatment is recommended for patients who do not respond to dietary and/or medication control of LDL cholesterol.

Enrollment: 62 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

Inclusion criteria

1. Men and women ≥18 years of age at the time of the screening visit
2. Diagnosis of HeFH (Heterozygous familial hypercholesterolemia)
3. 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

1. Homozygous FH (familial hypercholesterolemia)
2. Background medical LMT (lipid-modifying therapy) (if applicable) that has not been stable for at least 8 weeks prior to the screening visit
3. LDL apheresis schedule/ apheresis settings that have not been stable for at least 8 weeks prior to the screening visit
4. An LDL apheresis schedule other than QW to Q2W
5. Initiation of a new exercise program or exercise that has not remained stable within 8 weeks prior to the screening visit (week -2)
6. Initiation of a new diet or a diet that has not been stable within 8 weeks prior to the screening visit (week -2)
7. 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
8. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
9. Known history of a positive test for human immunodeficiency virus
10. Use of any active investigational drugs within 1 month or 5 half-lives of screening, whichever is longer
11. Patients who have been treated with at least 1 dose of alirocumab or any other anti-PCSK9 monoclonal antibody in any other clinical studies
12. Pregnant or breastfeeding women

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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62 participants in 3 patient groups

Placebo Q2W (Double Blind Period)

Experimental group

Description:

Placebo (for alirocumab) subcutaneous (SC) injection Q2W up to Week 16.

Treatment:

Drug: Placebo

Alirocumab 150 mg Q2W (Double Blind Period)

Experimental group

Description:

Alirocumab 150 mg SC injection Q2W up to Week 16.

Treatment:

Drug: Alirocumab

Alirocumab 150 Q2W (Open Label Treatment Period)

Experimental group

Description:

Alirocumab 150 mg SC injection Q2W starting from Week 18 up to Week 76.

Treatment:

Drug: Alirocumab