Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Phase 3

Completed

• Conditions: Homozygous Familial Hypercholesterolemia
• Interventions: Drug: evinacumab

• Registration Number: NCT03409744
• Lead Sponsor: Regeneron Pharmaceuticals

Brief Summary

The primary objectives of the study are:

* To evaluate the long-term safety and tolerability of evinacumab in patients with Homozygous Familial Hypercholesterolemia (HoFH)

* To evaluate the long-term safety and tolerability of evinacumab in adolescent patients with HoFH

The secondary objectives of the study are:

* To evaluate the effect of evinacumab on lipid parameters in patients with HoFH

* To evaluate the effect of evinacumab on lipid parameters in adolescent patients with HoFH

* To evaluate the potential development of anti-evinacumab antibodies

Detailed Description

Not available

Study Timeline

• Study First Submitted: 2018-01-08
• Study First Submitted QC: 2018-01-17
• Study First Posted: 2018-01-24
• Study Start: 2018-03-13
• Primary Completion: 2023-04-13
• Study Completion: 2023-04-13
• Results First Submitted: 2024-03-13
• Results First Submitted QC: 2024-04-26
• Results First Posted: 2024-04-30
• Status Verified: 2025-04
• Last Update Submitted: 2025-04-04
• Last Update Posted: 2025-04-08

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 116

Inclusion Criteria

1. Completion of the parent study in which they participated
2. Able to understand and complete study-related questionnaires

Key

Exclusion Criteria

1. Significant protocol deviation in the parent study based on the investigator's judgment, such as non-compliance by the patient
2. Concomitant medications that have not been stable prior to the baseline visit
3. Adverse event leading to permanent discontinuation from parent study
4. Any new condition or worsening of an existing condition, which in the opinion of the investigator would make the patient unsuitable for enrollment, or could interfere with the patient participating in or completing the study
5. Member of the clinical site study team and/or his/her immediate family
6. Pregnant or breastfeeding women
7. Women of childbearing potential who are unwilling to practice highly effective contraception prior to the initial dose/start of the first treatment, during the study, and for at least 24 weeks after the last dose of study drug
8. Men who are sexually active with women of childbearing potential and are unwilling to use the following forms of medically acceptable birth control during the study drug treatment period and for 24 weeks after the last injection of study drug: vasectomy with medical assessment of surgical success OR consistent use of a condom.

Note: Other protocol defined Inclusion/Exclusion criteria apply

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
evinacumab	evinacumab	-

Primary Outcome Measures

Name	Time	Method
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216	Up to 216 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.

Secondary Outcome Measures

Name	Time	Method
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Absolute Change in Non-HDL-C Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Absolute Change in LDL-C Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Percent Change in Triglycerides (TGs) Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Percent Change in Apolipoprotein B (Apo B) Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Percent Change in Total Cholesterol (TC) Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Absolute Change in Apo B Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Absolute Change in TC Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.
Absolute Change in TGs Over Time	Up to 120 weeks	The safety analysis set (SAF) included all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.

Trial Locations

Locations (5)

Clinical Trial Site #2; 🇺🇦 Kharkiv, Ukraine

Clinical Trial Site; 🇺🇦 Kyiv, Ukraine

Regeneron Study Site; 🇿🇦 Cape Town, Western Cape, South Africa

Clinical Trial Site #1; 🇺🇦 Kharkiv, Ukraine

Rgeneron Study Site; 🇺🇦 Kyiv, Ukraine