Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
Public ClinicalTrials.gov record NCT04233918. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
The primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH). The primary objective for Part B of the study is to demonstrate a reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab in pediatric (5 to 11 years of age) patients with HoFH. The secondary objective for Part A of the study is to evaluate the safety and tolerability of evinacumab administered intravenous (IV) in pediatric patients with HoFH. The secondary objectives for Part B of the study are: * To evaluate the effect of evinacumab on other lipid parameters (ie, apolipoprotein B (Apo B), non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol (TC), lipoprotein a \[Lp(a)\]) in pediatric patients with HoFH * To evaluate the safety and tolerability of evinacumab administered IV in pediatric patients with HoFH * To assess the PK of evinacumab in pediatric patients with HoFH * To assess the immunogenicity of evinacumab in pediatric patients with HoFH over time * To evaluate patient efficacy by mutation status
Study identification
- NCT ID
- NCT04233918
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 20 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 5 Years to 11 Years
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Jun 28, 2020
- Primary completion
- Jan 30, 2022
- Completion
- May 29, 2023
- Last update posted
- Jul 15, 2024
2020 – 2023
United States locations
- U.S. sites
- 6
- U.S. states
- 6
- U.S. cities
- 6
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Regeneron Research Site | Wilmington | Delaware | 19803 | — |
| Regeneron Research Site | Boca Raton | Florida | 33434 | — |
| Regeneron Research Site | Kansas City | Kansas | 66190 | — |
| Regeneron Research Center | Boston | Massachusetts | 02115 | — |
| Regeneron Research Center | Philadelphia | Pennsylvania | 19106 | — |
| Regeneron Research Center | Salt Lake City | Utah | 84108 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 5 non-U.S. sites.
Recently updated Homozygous Familial Hypercholesterolemia trials
Other public records listing Homozygous Familial Hypercholesterolemia. Sorted by last update posted; newest first. Not a recommendation.
- NCT07037771: A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE) Recruiting
- NCT01109368: The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository Recruiting
- NCT04815005: HoFH, the International Clinical Collaborators Registry Recruiting
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04233918, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jul 15, 2024 · Synced Sep 8, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04233918 live on ClinicalTrials.gov.