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

An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia

Status

Completed

Phases

Phase 3

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT03409744

Enrollment

116

Registered

2018-01-24

Start date

2018-03-13

Completion date

2023-04-13

Last updated

2025-04-08

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Homozygous Familial Hypercholesterolemia

Keywords

HoFH

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

Interventions

DRUGevinacumab

Intravenous (IV) administration

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

12 Years to No maximum

Healthy volunteers

Inclusion criteria

Key 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/

Design outcomes

Primary

Measure	Time frame	Description
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

Measure	Time frame	Description
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 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.
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.
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.
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 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.
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.
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.
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.

Countries

Australia, Austria, Canada, Czechia, France, Greece, Italy, Japan, Netherlands, South Africa, Ukraine, United States

Participant flow

Pre-assignment details

A total of 118 participants were planned to be enrolled. 116 participants were enrolled & treated. Reasons for screen fail were: 1 participant was unwilling to use protocol defined contraception, and 1 participant had a Low-density lipoprotein cholesterol (LDL-C) level less than the lower limit required for inclusion.

Participants by arm

Arm	Count
Total Evinacumab Includes all participants who were enrolled and received at least 1 dose or part of a dose of open-label study treatment.	116
Total	116

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Death	2
Overall Study	Lost to Follow-up	1
Overall Study	Physician Decision	5
Overall Study	Pregnancy	1
Overall Study	Protocol Violation	1

Baseline characteristics

Characteristic	Total Evinacumab
Age, Continuous	38.8 Years STANDARD_DEVIATION 15.92
Ethnicity (NIH/OMB) Hispanic or Latino	6 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	100 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	10 Participants
Race/Ethnicity, Customized American Indian or Alaska Native	0 Participants
Race/Ethnicity, Customized Asian	12 Participants
Race/Ethnicity, Customized Black or African American	4 Participants
Race/Ethnicity, Customized Native Hawaiian or Other Pacific Islander	0 Participants
Race/Ethnicity, Customized Not Reported	11 Participants
Race/Ethnicity, Customized Other	9 Participants
Race/Ethnicity, Customized White	80 Participants
Sex: Female, Male Female	57 Participants
Sex: Female, Male Male	59 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	2 / 116
other Total, other adverse events	79 / 116
serious Total, serious adverse events	27 / 116