ClinicalTrials.gg
Sign In
CompletedNCT03409744Updated Apr 8, 2025Results posted

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

A Phase 3 interventional study of evinacumab in Homozygous Familial Hypercholesterolemia, sponsored by Regeneron Pharmaceuticals. Completed at 38 sites in 12 countries. Open to participants aged 12 Years and older. Per ClinicalTrials.gov, last updated 2025-04-08.

Sponsored by Regeneron Pharmaceuticals · Phase 3, Interventional, and Treatment

Help build ClinicalTrials.gg — short surveys, remote interviews or testing, in your language. Take part →

Phase
Phase 3
Study type
Interventional
Enrollment
116
Allocation
Not applicable
Ages
12 Years and older
Sex
All
01

Where this study stands

Counted across the records on this site · 2026-11-14

Among 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States — 7 open to participants now, 9 under way, 4 of them phase 3. Homozygous Familial Hypercholesterolemia · United States.

Enrollment
6th of 31
116 · median 29
Started
2018
with 2 others
Phase 3
16 of 36
3 open now
Registered
20th of 36
by date
Lead sponsor
5 of 36
Regeneron Pharmaceuticals

Among the 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States, this one is the 6th largest by enrollment of the 31 interventional ones that give a count, is one of 3 that started in 2018, is one of 16 in phase 3, and was the 20th registered.

Homozygous Familial Hypercholesterolemia

68 studies on the registry are indexed under Homozygous Familial Hypercholesterolemia; 13 are open to participants now.

Browse Homozygous Familial Hypercholesterolemia studies →

Open to participants now

7 of the 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States are open now. Recruiting first, the newest first:

2 more — sign in →

All 7 open studies →

A study can be open while its sites in United States have not opened yet or have stopped. Only those recruiting at a site in United States →

Who these studies take

Of the 12 Homozygous Familial Hypercholesterolemia studies recruiting now, 6 (50%) set no upper age limit and 5 (42%) stop at 75 or younger; 8 take children under 18; none take healthy volunteers.

This study: 12 Years and older · all sexes · no healthy volunteers.

The full table on the hub →

This tab as a report to print or save as PDF → With an account.

Was this useful?
Watch this landscapeDownload CSVNarrow it in search →With an account: a weekly mail of what changed among 36 studies in Homozygous Familial Hypercholesterolemia with a site in United States, or all of them as a file.
02

Study 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
03

Conditions studied

  • Homozygous Familial Hypercholesterolemia

Keywords

  • HoFH
04

Who can participate

Ages eligible
12 Years and older
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility 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 who are unwilling to practice highly effective 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

Terms in these criteria, in plain language(2)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Childbearing potential

Able to become pregnant: from the first period until menopause, unless permanently unable to through surgery (removal of the womb, or of both ovaries or both fallopian tubes).

Criteria for people of childbearing potential usually ask for a negative pregnancy test and for contraception during the study and for a time after it.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

05

Study design

Phase
Phase 3
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrollment
116 participants (actual)

Study arms

  • Experimental
    evinacumab

    Drug: evinacumab

Interventions

  • Drugevinacumab

    Intravenous (IV) administration

    Also known as: REGN1500, EVKEEZA®

06

What researchers measure

Primary outcomes

  1. Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216

    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.

    Time frame: Up to 216 weeks

Secondary outcomes

  1. Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time

    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.

    Time frame: Up to 120 weeks

  2. Absolute Change in LDL-C Over Time

    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.

    Time frame: Up to 120 weeks

  3. Percent Change in Apolipoprotein B (Apo B) Over Time

    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.

    Time frame: Up to 120 weeks

  4. Absolute Change in Apo B Over Time

    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.

    Time frame: Up to 120 weeks

  5. Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time

    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.

    Time frame: Up to 120 weeks

  6. Absolute Change in Non-HDL-C Over Time

    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.

    Time frame: Up to 120 weeks

  7. Percent Change in Total Cholesterol (TC) Over Time

    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.

    Time frame: Up to 120 weeks

  8. Absolute Change in TC Over Time

    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.

    Time frame: Up to 120 weeks

  9. Percent Change in Triglycerides (TGs) Over Time

    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.

    Time frame: Up to 120 weeks

  10. Absolute Change in TGs Over Time

    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.

    Time frame: Up to 120 weeks

07

Results

Posted Apr 30, 2024

Participant flow

Participant flow — Overall Study
MilestoneTotal Evinacumab
Started116
Completed106
Not completed10
Withdrew: Death2
Withdrew: Physician decision5
Withdrew: Pregnancy1
Withdrew: Lost to follow-up1
Withdrew: Protocol violation1

Outcome measures

PrimaryNumber of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216

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.

Time frame:
Up to 216 weeks
Reported as:
Number · Number of participants
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216
Number of participantsOverall Study Total Evinacumab
Participants with any TEAE93
Participants with at least one serious TEAE27
SecondaryPercent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · Percentage of change
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time
Percentage of changeOverall Study Total Evinacumab
Week 8-46.45 ± 35.504
Week 24-43.64 ± 37.606
Week 48-43.88 ± 36.037
Week 72-45.17 ± 31.571
Week 96-38.00 ± 52.859
Week 120-33.10 ± 65.888
SecondaryAbsolute Change in LDL-C Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · mg/dL
Absolute Change in LDL-C Over Time
mg/dLOverall Study Total Evinacumab
Week 8-142.1 ± 114.61
Week 24-132.0 ± 124.37
Week 48-132.8 ± 133.29
Week 72-135.1 ± 121.91
Week 96-131.4 ± 129.29
Week 120-132.5 ± 132.03
SecondaryPercent Change in Apolipoprotein B (Apo B) Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · Percentage of change
Percent Change in Apolipoprotein B (Apo B) Over Time
Percentage of changeOverall Study Total Evinacumab
Week 8-39.74 ± 25.338
Week 24-36.98 ± 27.574
Week 48-35.85 ± 29.998
Week 72-37.60 ± 27.292
Week 96-32.54 ± 39.969
Week 120-27.73 ± 51.722
SecondaryAbsolute Change in Apo B Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · mg/dL
Absolute Change in Apo B Over Time
mg/dLOverall Study Total Evinacumab
Week 8-78.0 ± 59.78
Week 24-70.5 ± 61.96
Week 48-69.4 ± 69.95
Week 72-73.9 ± 61.51
Week 96-71.5 ± 66.66
Week 120-70.7 ± 76.05
SecondaryPercent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · Percentage of change
Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time
Percentage of changeOverall Study Total Evinacumab
Week 8-48.47 ± 28.369
Week 24-46.14 ± 29.117
Week 48-45.15 ± 33.566
Week 72-46.69 ± 28.534
Week 96-40.88 ± 46.588
Week 120-35.85 ± 58.374
SecondaryAbsolute Change in Non-HDL-C Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · mg/dL
Absolute Change in Non-HDL-C Over Time
mg/dLOverall Study Total Evinacumab
Week 8-153.7 ± 115.66
Week 24-143.9 ± 125.03
Week 48-144.1 ± 136.95
Week 72-147.3 ± 122.76
Week 96-144.5 ± 131.15
Week 120-142.9 ± 136.65
SecondaryPercent Change in Total Cholesterol (TC) Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · Percentage of change
Percent Change in Total Cholesterol (TC) Over Time
Percentage of changeOverall Study Total Evinacumab
Week 8-47.04 ± 20.722
Week 24-44.17 ± 24.224
Week 48-43.78 ± 27.838
Week 72-44.58 ± 25.700
Week 96-40.33 ± 38.163
Week 120-36.92 ± 47.469
SecondaryAbsolute Change in TC Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · mg/dL
Absolute Change in TC Over Time
mg/dLOverall Study Total Evinacumab
Week 8-167.3 ± 115.76
Week 24-157.4 ± 125.93
Week 48-158.2 ± 136.51
Week 72-160.7 ± 123.09
Week 96-157.2 ± 132.40
Week 120-153.8 ± 138.26
SecondaryPercent Change in Triglycerides (TGs) Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · Percentage of change
Percent Change in Triglycerides (TGs) Over Time
Percentage of changeOverall Study Total Evinacumab
Week 8-45.97 ± 26.024
Week 24-46.93 ± 24.582
Week 48-43.25 ± 37.443
Week 72-46.50 ± 25.826
Week 96-48.28 ± 24.136
Week 120-42.06 ± 36.945
SecondaryAbsolute Change in TGs Over Time

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.

Time frame:
Up to 120 weeks
Reported as:
Mean · mg/dL
Absolute Change in TGs Over Time
mg/dLOverall Study Total Evinacumab
Week 8-64.4 ± 89.77
Week 24-68.1 ± 97.29
Week 48-66.7 ± 97.02
Week 72-68.8 ± 91.52
Week 96-75.2 ± 107.49
Week 120-66.7 ± 110.95

Adverse events

Collected over From signing of Informed Consent to week 216. Non-serious events are listed at a 5% frequency threshold.

Adverse event summary by group
GroupDeathsSeriousOther
Overall2/116 (1.7%)27/116 (23.3%)79/116 (68.1%)
Most frequent serious events
Showing 10 of 38
Most frequent serious events
EventOverall
Angina pectorisCardiac disorders2/116
Angina unstableCardiac disorders2/116
Aortic valve diseaseCardiac disorders2/116
Coronary artery diseaseCardiac disorders2/116
Chest painGeneral disorders2/116
Acute myocardial infarctionCardiac disorders1/116
Atrial fibrillationCardiac disorders1/116
Cardiac arrestCardiac disorders1/116
Cardiac failure acuteCardiac disorders1/116
Cardiac failure chronicCardiac disorders1/116
Most frequent other events
Showing 10 of 22
Most frequent other events
EventOverall
NasopharyngitisInfections and infestations23/116
COVID-19Infections and infestations19/116
HeadacheNervous system disorders19/116
Influenza like illnessGeneral disorders17/116
ArthralgiaMusculoskeletal and connective tissue disorders15/116
NauseaGastrointestinal disorders14/116
Back painMusculoskeletal and connective tissue disorders14/116
CoughRespiratory, thoracic and mediastinal disorders12/116
GastroenteritisInfections and infestations10/116
Urinary tract infectionInfections and infestations10/116

Baseline characteristics

Age, Continuous
Age, Continuous(Years)Total Evinacumab
Mean38.8 ± 15.92
Sex: Female, Male
Sex: Female, Male(Participants)Total Evinacumab
Female57
Male59
Ethnicity (NIH/OMB)
Ethnicity (NIH/OMB)(Participants)Total Evinacumab
Hispanic or Latino6
Not Hispanic or Latino100
Unknown or Not Reported10
Race/Ethnicity, Customized
Race/Ethnicity, Customized(Participants)Total Evinacumab
White80
Black or African American4
Asian12
American Indian or Alaska Native0
Native Hawaiian or Other Pacific Islander0
Not Reported11
Other9
08

Study locations

38 sites
  • Clinical Trial Site
    Los Angeles, California 90048, United States
  • Clinical Trial Site
    Boca Raton, Florida 33434, United States
  • Clinical Trial Site
    Boston, Massachusetts 02114, United States
  • Clinical Trial Site
    New York, New York 10029, United States
  • Clinical Trial Site
    Cincinnati, Ohio 45227, United States
  • Clinical Trial Site
    Portland, Oregon 97239, United States
  • Clinical Trial Site
    Philadelphia, Pennsylvania 19104, United States
  • Clinical Trial Site
    Dallas, Texas 75226, United States
  • Clinical Trial Site
    Dallas, Texas 75390, United States
  • Clinical Trial Site
    Camperdown, New South Wales 2050, Australia
  • Clinical Trial Site
    Perth, Western Australia 6000, Australia
  • Regeneron Study Site
    Vienna, Wien 1090, Austria
  • Regeneron Study Site
    Innsbruck, 6020, Austria
  • Clinical Trial Site
    Chicoutimi, Quebec G7H 7K9, Canada
  • Clinical Trial Site
    Quebec, G1V 4W2, Canada
  • Regeneron Study Site
    Praha 2, Kateřinská 121 08, Czechia
  • Clinical Trial Site
    Paris, Cedex 13 75651, France
  • Clinical Trial Site
    Marseille, Cedex 5 13385, France
  • Clinical Trial Site
    Dijon, Cedex 21079, France
  • Regeneron Study Site
    Kallithea, Athens 17674, Greece
  • Clinical Trial Site
    Ioannina, 45500, Greece
  • Clinical Trial Site
    Napoli, Campania 80131, Italy
  • Clinical Trial Site
    Roma, Rome 161, Italy
  • Clinical Trial Site
    Obu, Aichi 474-8710, Japan
  • Regeneron Study Site
    Kurume, Fukuoka 830-8522, Japan
  • Regeneron Study Site
    Nishinomiya, Hyogo 662-0918, Japan
  • Regeneron Study Site
    Kanazawa, Ishikawa 920-8641, Japan
  • Regeneron Study Site
    Kita-ku, Osaka 530-0001, Japan
  • Regeneron Study Site
    Suita, Osaka 564-8565, Japan
  • Regeneron Study Site
    Suita, Osaka 565-0871, Japan
  • Clinical Trial Site
    Amsterdam, 1105 AZ, Netherlands
  • Regeneron Study Site
    Johannesburg, Gauteng 2000, South Africa
  • Regeneron Study Site
    Cape Town, Western Cape 7925, South Africa
  • Clinical Trial Site
    Ivano-Frankivsk, 76075, Ukraine
  • Clinical Trial Site #1
    Kharkiv, 61039, Ukraine
  • Clinical Trial Site #2
    Kharkiv, 61176, Ukraine
  • Clinical Trial Site
    Kyiv, 02660, Ukraine
  • Rgeneron Study Site
    Kyiv, 3680, Ukraine
09

References and documents

Study documents

PDFs hosted by the registry, ClinicalTrials.gov.

10

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reachedSimilar studies: half by the dot; the band is the middle halfHalf of similar studies reached it by the dot; the band is the middle half, from a quarter to three quarters

Ran from Mar 2018 to primary completion in Apr 2023, 61 months.

Results were first posted in Apr 2024, 1 year after primary completion.

Of 20 phase 3 interventional studies under Homozygous Familial Hypercholesterolemia, half reached primary completion within 22 months of starting (the median), counting those still running; a quarter within 16 and three quarters within 34. This study's 61 months is above that.

How these figures are worked out, and which studies are left out of them.

Registered
Jan 8, 2018
First posted
Jan 24, 2018
StartActual
Mar 13, 2018
Primary completionActual
Apr 13, 2023
CompletionActual
Apr 13, 2023
Results first posted
Apr 30, 2024
Last update
Apr 8, 2025
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Apr 8, 2025, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
11

Registry details

Key details

Study ID
NCT03409744
Lead sponsor
Regeneron Pharmaceuticals
Responsible party
Sponsor
First posted
Jan 24, 2018
Start date
Mar 13, 2018
Primary completion
Apr 13, 2023
Completion
Apr 13, 2023
Results posted
Apr 30, 2024
Last update
Apr 8, 2025

Study contacts

Clinical Trial Management
study director · Regeneron Pharmaceuticals

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is completed, as verified in Apr 2025. You cannot join it, but the record below documents what was studied.

Follow this study

Get an email when the registry record changes — status, dates, results — or when someone posts here.

Sign in to follow

Discussion

Questions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.

Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.

Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.

Studies of the drug tested here: Evinacumab

Other studies in this condition

Homozygous Familial Hypercholesterolemia →

Other studies from this sponsor

Regeneron Pharmaceuticals →

Start the discussion