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

Homozygous Familial Hypercholesterolemia Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03409744
• Other study ID #: R1500-CL-1719
• Secondary ID: 2017-003170-13
• Status: Completed
• Phase: Phase 3
• Start date: March 13, 2018
• Est. completion date: April 13, 2023

Study information

• Verified date: April 2024
• Source: Regeneron Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical 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 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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 116
• Est. completion date: April 13, 2023
• Est. primary completion date: April 13, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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/Exclusion criteria apply

Related Conditions & MeSH terms

• Homozygous Familial Hypercholesterolemia
• Hypercholesterolemia
• Hyperlipoproteinemia Type II

Intervention

Drug:
• evinacumab
Intravenous (IV) administration

Locations

Country	Name	City	State
Australia	Clinical Trial Site	Camperdown	New South Wales
Australia	Clinical Trial Site	Perth	Western Australia
Austria	Regeneron Study Site	Innsbruck
Austria	Regeneron Study Site	Vienna	Wien
Canada	Clinical Trial Site	Chicoutimi	Quebec
Canada	Clinical Trial Site	Quebec
Czechia	Regeneron Study Site	Praha 2	Katerinská
France	Clinical Trial Site	Dijon	Cedex
France	Clinical Trial Site	Marseille	Cedex 5
France	Clinical Trial Site	Paris	Cedex 13
Greece	Clinical Trial Site	Ioannina
Greece	Regeneron Study Site	Kallithea	Athens
Italy	Clinical Trial Site	Napoli	Campania
Italy	Clinical Trial Site	Roma	Rome
Japan	Regeneron Study Site	Kanazawa	Ishikawa
Japan	Regeneron Study Site	Kita-ku	Osaka
Japan	Regeneron Study Site	Kurume	Fukuoka
Japan	Regeneron Study Site	Nishinomiya	Hyogo
Japan	Clinical Trial Site	Obu	Aichi
Japan	Regeneron Study Site	Suita	Osaka
Japan	Regeneron Study Site	Suita	Osaka
Netherlands	Clinical Trial Site	Amsterdam
South Africa	Regeneron Study Site	Cape Town	Western Cape
South Africa	Regeneron Study Site	Johannesburg	Gauteng
Ukraine	Clinical Trial Site	Ivano-Frankivsk
Ukraine	Clinical Trial Site #1	Kharkiv
Ukraine	Clinical Trial Site #2	Kharkiv
Ukraine	Clinical Trial Site	Kyiv
Ukraine	Rgeneron Study Site	Kyiv
United States	Clinical Trial Site	Boca Raton	Florida
United States	Clinical Trial Site	Boston	Massachusetts
United States	Clinical Trial Site	Cincinnati	Ohio
United States	Clinical Trial Site	Dallas	Texas
United States	Clinical Trial Site	Dallas	Texas
United States	Clinical Trial Site	Los Angeles	California
United States	Clinical Trial Site	New York	New York
United States	Clinical Trial Site	Philadelphia	Pennsylvania
United States	Clinical Trial Site	Portland	Oregon

Sponsors (1)

Lead Sponsor	Collaborator
Regeneron Pharmaceuticals

Countries where clinical trial is conducted

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

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	Up to 216 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks
Secondary	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.	Up to 120 weeks