Homozygous Familial HypercholesterolemiaPhase 3NCT03409744
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
A Phase 3 study testing evinacumab in people with Homozygous Familial Hypercholesterolemia, run by Regeneron Pharmaceuticals at 38 sites in 12 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Homozygous Familial Hypercholesterolemia
- What is being tested?
- evinacumab
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 116 participants
- Where
- 38 sitesin 12 countries
- Study dates
- 13 Mar 2018to 13 Apr 2023
- Phase
- Phase 3
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Completion of the parent study in which they participated
- Able to understand and complete study-related questionnaires
Who cannot join
- Significant protocol deviation in the parent study based on the investigator's judgment, such as non-compliance by the patient
- Concomitant medications that have not been stable prior to the baseline visit
- Adverse event leading to permanent discontinuation from parent study
- 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
- Member of the clinical site study team and/or his/her immediate family
- Pregnant or breastfeeding women
Show all 9Show fewer
- 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
- 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
Trial locations and contacts
38 sites in 12 countries.
38 sites in 12 countries
United States9 sites
Clinical Trial Site
Boca Raton, Florida, 33434
Clinical Trial Site
Boston, Massachusetts, 02114
Clinical Trial Site
Cincinnati, Ohio, 45227
Clinical Trial Site
Dallas, Texas, 75226
Clinical Trial Site
Dallas, Texas, 75390
Clinical Trial Site
Los Angeles, California, 90048
Clinical Trial Site
New York, New York, 10029
Clinical Trial Site
Philadelphia, Pennsylvania, 19104
Clinical Trial Site
Portland, Oregon, 97239
Japan7 sites
Regeneron Study Site
Kanazawa, Ishikawa-ken, 920-8641
Regeneron Study Site
Kita-ku, Osaka, 530-0001
Regeneron Study Site
Kurume, Fukuoka, 830-8522
Regeneron Study Site
Nishinomiya, Hyōgo, 662-0918
Clinical Trial Site
Ōbu, Aichi-ken, 474-8710
Regeneron Study Site
Suita, Osaka, 564-8565
Regeneron Study Site
Suita, Osaka, 565-0871
Ukraine5 sites
Clinical Trial Site
Ivano-Frankivsk, 76075
Clinical Trial Site #1
Kharkiv, 61039
Clinical Trial Site #2
Kharkiv, 61176
Clinical Trial Site
Kyiv, 02660
Rgeneron Study Site
Kyiv, 3680
France3 sites
Clinical Trial Site
Dijon, Cedex, 21079
Clinical Trial Site
Marseille, Cedex 5, 13385
Clinical Trial Site
Paris, Cedex 13, 75651
Australia2 sites
Clinical Trial Site
Camperdown, New South Wales, 2050
Clinical Trial Site
Perth, Western Australia, 6000
Austria2 sites
Regeneron Study Site
Innsbruck, 6020
Regeneron Study Site
Vienna, State of Vienna, 1090
Canada2 sites
Clinical Trial Site
Chicoutimi, Quebec, G7H 7K9
Clinical Trial Site
Québec, G1V 4W2
Greece2 sites
Clinical Trial Site
Ioannina, 45500
Regeneron Study Site
Kallithea, Athens, 17674
Italy2 sites
Clinical Trial Site
Naples, Campania, 80131
Clinical Trial Site
Roma, Rome, 161
South Africa2 sites
Regeneron Study Site
Cape Town, Western Cape, 7925
Regeneron Study Site
Johannesburg, Gauteng, 2000
About this study
Official title: An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
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
- Conditions
- Homozygous Familial Hypercholesterolemia
- Being tested
- evinacumab
- Keywords
- HoFH
- Medical subject headings
- Homozygous Familial Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
evinacumabDrug
Also called REGN1500, EVKEEZA®
Intravenous (IV) administration
| Study group | Role | What they receive |
|---|---|---|
| evinacumab | Experimental | Not described Receives: Drug: evinacumab |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Up to 216 weeks
Secondary outcomes (10)
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
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.
Measured: Up to 120 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Jan 2018
- Study start13 Mar 2018
- Primary completion13 Apr 2023
- Study completion13 Apr 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Homozygous Familial HypercholesterolemiaStudies of the same condition, open and under way.
- More trials run by Regeneron PharmaceuticalsThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 116 participants (actual)
Sponsor and investigators
- Lead sponsor
- Regeneron Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Clinical Trial Management, Study director, Regeneron Pharmaceuticals
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03409744
- Sponsor's protocol ID
- R1500-CL-1719
- Other IDs
- 2017-003170-13, 2017
- Status last verified
- Apr 2025
- First submitted
- 8 Jan 2018
- Last updated
- 8 Apr 2025
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 8 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
- ClinicalTrials.gov ID
- NCT03409744
- Acronym / sponsor ID
- R1500-CL-1719
- Conditions
- Homozygous Familial Hypercholesterolemia
- Keywords
- HoFH
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 116 participants (actual)
- Lead sponsor
- Regeneron PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 13 Mar 2018 (actual)
- Primary completion
- 13 Apr 2023 (actual)
- Study completion
- 13 Apr 2023 (actual)
- First posted
- 24 Jan 2018 (actual)
- Results
- Posted 30 Apr 2024
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| evinacumab | Experimental | Drug: evinacumab |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Completion of the parent study in which they participated
- Able to understand and complete study-related questionnaires
Key exclusion criteria (9 listed)
- Significant protocol deviation in the parent study based on the investigator's judgment, such as non-compliance by the patient
- Concomitant medications that have not been stable prior to the baseline visit
- Adverse event leading to permanent discontinuation from parent study
- 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
- Member of the clinical site study team and/or his/her immediate family
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| evinacumabAlso: REGN1500, EVKEEZA®Intravenous (IV) administration | Drug | evinacumab |
Primary outcome
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216
Time frame: 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.
10 secondary outcomes
Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time
Time frame: Up to 120 weeks
Absolute Change in LDL-C Over Time
Time frame: Up to 120 weeks
Percent Change in Apolipoprotein B (Apo B) Over Time
Time frame: Up to 120 weeks
Absolute Change in Apo B Over Time
Time frame: Up to 120 weeks
Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time
Time frame: Up to 120 weeks
Absolute Change in Non-HDL-C Over Time
Time frame: Up to 120 weeks
Percent Change in Total Cholesterol (TC) Over Time
Time frame: Up to 120 weeks
Absolute Change in TC Over Time
Time frame: Up to 120 weeks
Percent Change in Triglycerides (TGs) Over Time
Time frame: Up to 120 weeks
Absolute Change in TGs Over Time
Time frame: Up to 120 weeks
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 8 Apr 2025.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- A Plain Language Summary is available on TrialSummaries.comLink provided by the sponsor.
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Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
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Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Pediatric ORION-16, ORION-13, ORION-20, or ORION-19 Studies
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A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Clinical Trial Management study director
Regeneron Pharmaceuticals
38 sites in 12 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing evinacumab in people with Homozygous Familial Hypercholesterolemia, run by Regeneron Pharmaceuticals at 38 sites in 12 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 2 inclusion and 9 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 8 Apr 2025. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 38 sites in United States, Japan, Ukraine, France, Australia and 7 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 30 Apr 2024). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216 (time frame: Up to 216 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 8 Apr 2025.