Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Primary Purpose
Homozygous Familial Hypercholesterolemia
Status
Completed
Phase
Phase 3
Locations
International
Study Type
Interventional
Intervention
evinacumab
Sponsored by
About this trial
This is an interventional treatment trial for Homozygous Familial Hypercholesterolemia focused on measuring HoFH
Eligibility Criteria
Key Inclusion Criteria:
- Completion of the parent study in which they participated
- Able to understand and complete study-related questionnaires
Key Exclusion Criteria:
- 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
- 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
Sites / Locations
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Regeneron Study Site
- Regeneron Study Site
- Clinical Trial Site
- Clinical Trial Site
- Regeneron Study Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Regeneron Study Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Clinical Trial Site
- Regeneron Study Site
- Regeneron Study Site
- Regeneron Study Site
- Regeneron Study Site
- Regeneron Study Site
- Regeneron Study Site
- Clinical Trial Site
- Regeneron Study Site
- Regeneron Study Site
- Clinical Trial Site
- Clinical Trial Site #1
- Clinical Trial Site #2
- Clinical Trial Site
- Rgeneron Study Site
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
evinacumab
Arm Description
Outcomes
Primary Outcome Measures
Incidence and severity of treatment-emergent adverse events (TEAEs)
Secondary Outcome Measures
Percent change in Low-Density Lipoprotein Cholesterol (LDL-C) over time
Absolute change in LDL-C over time
Percent change in Apolipoprotein B (Apo B) over time
Absolute change in Apo B over time
Percent change in non-High-Density Lipoprotein Cholesterol (HDL-C) over time
Absolute change in non-HDL-C over time
Percent change in Total Cholesterol (TC) over time
Absolute change in TC over time
Percent change in Triglycerides (TGs) over time
Absolute change in TGs over time
Presence of anti-evinacumab antibodies
Full Information
1. Study Identification
Unique Protocol Identification Number
NCT03409744
Brief Title
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Official Title
An Open-Label Study to Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Study Type
Interventional
2. Study Status
Record Verification Date
April 2023
Overall Recruitment Status
Completed
Study Start Date
March 13, 2018 (Actual)
Primary Completion Date
April 13, 2023 (Actual)
Study Completion Date
April 13, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Regeneron Pharmaceuticals
4. Oversight
Studies a U.S. FDA-regulated Drug Product
Yes
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Homozygous Familial Hypercholesterolemia
Keywords
HoFH
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 3
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
116 (Actual)
8. Arms, Groups, and Interventions
Arm Title
evinacumab
Arm Type
Experimental
Intervention Type
Drug
Intervention Name(s)
evinacumab
Other Intervention Name(s)
REGN1500, EVKEEZA®
Intervention Description
Intravenous (IV) administration
Primary Outcome Measure Information:
Title
Incidence and severity of treatment-emergent adverse events (TEAEs)
Time Frame
Up to 192 weeks
Secondary Outcome Measure Information:
Title
Percent change in Low-Density Lipoprotein Cholesterol (LDL-C) over time
Time Frame
Up to 192 weeks
Title
Absolute change in LDL-C over time
Time Frame
Up to 192 weeks
Title
Percent change in Apolipoprotein B (Apo B) over time
Time Frame
Up to 192 weeks
Title
Absolute change in Apo B over time
Time Frame
Up to 192 weeks
Title
Percent change in non-High-Density Lipoprotein Cholesterol (HDL-C) over time
Time Frame
Up to 192 weeks
Title
Absolute change in non-HDL-C over time
Time Frame
Up to 192 weeks
Title
Percent change in Total Cholesterol (TC) over time
Time Frame
Up to 192 weeks
Title
Absolute change in TC over time
Time Frame
Up to 192 weeks
Title
Percent change in Triglycerides (TGs) over time
Time Frame
Up to 192 weeks
Title
Absolute change in TGs over time
Time Frame
Up to 192 weeks
Title
Presence of anti-evinacumab antibodies
Time Frame
Up to 192 weeks
10. Eligibility
Sex
All
Minimum Age & Unit of Time
12 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Key Inclusion Criteria:
Completion of the parent study in which they participated
Able to understand and complete study-related questionnaires
Key Exclusion Criteria:
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
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Clinical Trial Management
Organizational Affiliation
Regeneron Pharmaceuticals
Official's Role
Study Director
Facility Information:
Facility Name
Clinical Trial Site
City
Los Angeles
State/Province
California
ZIP/Postal Code
90048
Country
United States
Facility Name
Clinical Trial Site
City
Boca Raton
State/Province
Florida
ZIP/Postal Code
33434
Country
United States
Facility Name
Clinical Trial Site
City
Boston
State/Province
Massachusetts
ZIP/Postal Code
02114
Country
United States
Facility Name
Clinical Trial Site
City
New York
State/Province
New York
ZIP/Postal Code
10029
Country
United States
Facility Name
Clinical Trial Site
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45227
Country
United States
Facility Name
Clinical Trial Site
City
Portland
State/Province
Oregon
ZIP/Postal Code
97239
Country
United States
Facility Name
Clinical Trial Site
City
Philadelphia
State/Province
Pennsylvania
ZIP/Postal Code
19104
Country
United States
Facility Name
Clinical Trial Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75226
Country
United States
Facility Name
Clinical Trial Site
City
Dallas
State/Province
Texas
ZIP/Postal Code
75390
Country
United States
Facility Name
Clinical Trial Site
City
Camperdown
State/Province
New South Wales
ZIP/Postal Code
2050
Country
Australia
Facility Name
Clinical Trial Site
City
Perth
State/Province
Western Australia
ZIP/Postal Code
6000
Country
Australia
Facility Name
Regeneron Study Site
City
Vienna
State/Province
Wien
ZIP/Postal Code
1090
Country
Austria
Facility Name
Regeneron Study Site
City
Innsbruck
ZIP/Postal Code
6020
Country
Austria
Facility Name
Clinical Trial Site
City
Chicoutimi
State/Province
Quebec
ZIP/Postal Code
G7H 7K9
Country
Canada
Facility Name
Clinical Trial Site
City
Quebec
ZIP/Postal Code
G1V 4W2
Country
Canada
Facility Name
Regeneron Study Site
City
Praha 2
State/Province
Kateřinská
ZIP/Postal Code
121 08
Country
Czechia
Facility Name
Clinical Trial Site
City
Paris
State/Province
Cedex 13
ZIP/Postal Code
75651
Country
France
Facility Name
Clinical Trial Site
City
Marseille
State/Province
Cedex 5
ZIP/Postal Code
13385
Country
France
Facility Name
Clinical Trial Site
City
Dijon
State/Province
Cedex
ZIP/Postal Code
21079
Country
France
Facility Name
Regeneron Study Site
City
Kallithea
State/Province
Athens
ZIP/Postal Code
17674
Country
Greece
Facility Name
Clinical Trial Site
City
Ioannina
ZIP/Postal Code
45500
Country
Greece
Facility Name
Clinical Trial Site
City
Napoli
State/Province
Campania
ZIP/Postal Code
80131
Country
Italy
Facility Name
Clinical Trial Site
City
Roma
State/Province
Rome
ZIP/Postal Code
161
Country
Italy
Facility Name
Clinical Trial Site
City
Obu
State/Province
Aichi
ZIP/Postal Code
474-8710
Country
Japan
Facility Name
Regeneron Study Site
City
Kurume
State/Province
Fukuoka
ZIP/Postal Code
830-8522
Country
Japan
Facility Name
Regeneron Study Site
City
Nishinomiya
State/Province
Hyogo
ZIP/Postal Code
662-0918
Country
Japan
Facility Name
Regeneron Study Site
City
Kanazawa
State/Province
Ishikawa
ZIP/Postal Code
920-8641
Country
Japan
Facility Name
Regeneron Study Site
City
Kita-ku
State/Province
Osaka
ZIP/Postal Code
530-0001
Country
Japan
Facility Name
Regeneron Study Site
City
Suita
State/Province
Osaka
ZIP/Postal Code
564-8565
Country
Japan
Facility Name
Regeneron Study Site
City
Suita
State/Province
Osaka
ZIP/Postal Code
565-0871
Country
Japan
Facility Name
Clinical Trial Site
City
Amsterdam
ZIP/Postal Code
1105 AZ
Country
Netherlands
Facility Name
Regeneron Study Site
City
Johannesburg
State/Province
Gauteng
ZIP/Postal Code
2000
Country
South Africa
Facility Name
Regeneron Study Site
City
Cape Town
State/Province
Western Cape
ZIP/Postal Code
7925
Country
South Africa
Facility Name
Clinical Trial Site
City
Ivano-Frankivsk
ZIP/Postal Code
76075
Country
Ukraine
Facility Name
Clinical Trial Site #1
City
Kharkiv
ZIP/Postal Code
61039
Country
Ukraine
Facility Name
Clinical Trial Site #2
City
Kharkiv
ZIP/Postal Code
61176
Country
Ukraine
Facility Name
Clinical Trial Site
City
Kyiv
ZIP/Postal Code
02660
Country
Ukraine
Facility Name
Rgeneron Study Site
City
Kyiv
ZIP/Postal Code
3680
Country
Ukraine
12. IPD Sharing Statement
Learn more about this trial
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
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