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

NCT ID: NCT02326220

Last Updated: 2020-05-01

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 62 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2015-03-31
• Study Completion Date: 2016-04-30

Brief 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.

Detailed 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.

Conditions

Heterozygous Familial Hypercholesterolemia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Placebo Q2W (Double Blind Period)

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

Group Type: EXPERIMENTAL

Placebo

Intervention Type: DRUG

Alirocumab 150 mg Q2W (Double Blind Period)

Alirocumab 150 mg SC injection Q2W up to Week 16.

Group Type: EXPERIMENTAL

Alirocumab

Intervention Type: DRUG

Alirocumab 150 Q2W (Open Label Treatment Period)

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

Group Type: EXPERIMENTAL

Alirocumab

Intervention Type: DRUG

Interventions

Placebo

Intervention Type: DRUG

Alirocumab

Intervention Type: DRUG

Other Intervention Names

REGN727; SAR236553; Praluent®

Eligibility Criteria

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Sanofi

INDUSTRY

Sponsor Role: collaborator

Regeneron Pharmaceuticals

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Clinical Trial Management

Role: STUDY_DIRECTOR

Regeneron Pharmaceuticals

Locations

Aurora, Colorado, United States

Hartford, Connecticut, United States

Kansas City, Kansas, United States

Scarborough, Maine, United States

Rochester, Minnesota, United States

Portland, Oregon, United States

Philadelphia, Pennsylvania, United States

Dresden, Saxony, Germany

Berlin, , Germany

Göttingen, , Germany

Muenchen (2 Locations), , Germany

Passau, , Germany

Rostock, , Germany

Countries

United States; Germany

References

Moriarty PM, Parhofer KG, Babirak SP, Cornier MA, Duell PB, Hohenstein B, Leebmann J, Ramlow W, Schettler V, Simha V, Steinhagen-Thiessen E, Thompson PD, Vogt A, von Stritzky B, Du Y, Manvelian G. Alirocumab in patients with heterozygous familial hypercholesterolaemia undergoing lipoprotein apheresis: the ODYSSEY ESCAPE trial. Eur Heart J. 2016 Dec 21;37(48):3588-3595. doi: 10.1093/eurheartj/ehw388. Epub 2016 Aug 29.

Reference Type: BACKGROUND

PMID: 27572070 (View on PubMed)

Moriarty PM, Parhofer KG, Babirak SP, deGoma E, Duell PB, Hohenstein B, Ramlow W, Simha V, Steinhagen-Thiessen E, Thompson PD, Vogt A, von Stritzky B, Du Y, Manvelian G. Alirocumab in patients with heterozygous familial hypercholesterolemia undergoing lipoprotein apheresis: Rationale and design of the ODYSSEY ESCAPE trial. J Clin Lipidol. 2016 May-Jun;10(3):627-34. doi: 10.1016/j.jacl.2016.02.003. Epub 2016 Feb 18.

Reference Type: DERIVED

PMID: 27206951 (View on PubMed)

Other Identifiers

R727-CL-1216

Identifier Type: -

Identifier Source: org_study_id