Clinical trial NCT03409744: 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
Overall status: COMPLETED
Phase: PHASE3
Conditions: Homozygous Familial Hypercholesterolemia
Keywords: HoFH
Lead sponsor: Regeneron Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2018-01-24
Enrollment: 116
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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


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 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

Primary outcomes:
measure: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216
timeFrame: Up to 216 weeks
Secondary outcomes:
measure: Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time
timeFrame: Up to 120 weeks
measure: Absolute Change in LDL-C Over Time
timeFrame: Up to 120 weeks
measure: Percent Change in Apolipoprotein B (Apo B) Over Time
timeFrame: Up to 120 weeks
measure: Absolute Change in Apo B Over Time
timeFrame: Up to 120 weeks
measure: Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time
timeFrame: Up to 120 weeks
measure: Absolute Change in Non-HDL-C Over Time
timeFrame: Up to 120 weeks
measure: Percent Change in Total Cholesterol (TC) Over Time
timeFrame: Up to 120 weeks
measure: Absolute Change in TC Over Time
timeFrame: Up to 120 weeks
measure: Percent Change in Triglycerides (TGs) Over Time
timeFrame: Up to 120 weeks
measure: Absolute Change in TGs Over Time
timeFrame: Up to 120 weeks
Other outcomes:
Locations:
city: Los Angeles
country: United States
facility: Clinical Trial Site
state: California
city: Boca Raton
country: United States
facility: Clinical Trial Site
state: Florida
city: Boston
country: United States
facility: Clinical Trial Site
state: Massachusetts
city: New York
country: United States
facility: Clinical Trial Site
state: New York
city: Cincinnati
country: United States
facility: Clinical Trial Site
state: Ohio
city: Portland
country: United States
facility: Clinical Trial Site
state: Oregon
city: Philadelphia
country: United States
facility: Clinical Trial Site
state: Pennsylvania
city: Dallas
country: United States
facility: Clinical Trial Site
state: Texas
city: Dallas
country: United States
facility: Clinical Trial Site
state: Texas
city: Camperdown
country: Australia
facility: Clinical Trial Site
state: New South Wales
city: Perth
country: Australia
facility: Clinical Trial Site
state: Western Australia
city: Vienna
country: Austria
facility: Regeneron Study Site
state: Wien
city: Innsbruck
country: Austria
facility: Regeneron Study Site
city: Chicoutimi
country: Canada
facility: Clinical Trial Site
state: Quebec
city: Quebec
country: Canada
facility: Clinical Trial Site
city: Praha 2
country: Czechia
facility: Regeneron Study Site
state: Kateřinská
city: Paris
country: France
facility: Clinical Trial Site
state: Cedex 13
city: Marseille
country: France
facility: Clinical Trial Site
state: Cedex 5
city: Dijon
country: France
facility: Clinical Trial Site
state: Cedex
city: Kallithea
country: Greece
facility: Regeneron Study Site
state: Athens
city: Ioannina
country: Greece
facility: Clinical Trial Site
city: Napoli
country: Italy
facility: Clinical Trial Site
state: Campania
city: Roma
country: Italy
facility: Clinical Trial Site
state: Rome
city: Obu
country: Japan
facility: Clinical Trial Site
state: Aichi
city: Kurume
country: Japan
facility: Regeneron Study Site
state: Fukuoka
city: Nishinomiya
country: Japan
facility: Regeneron Study Site
state: Hyogo
city: Kanazawa
country: Japan
facility: Regeneron Study Site
state: Ishikawa
city: Kita-ku
country: Japan
facility: Regeneron Study Site
state: Osaka
city: Suita
country: Japan
facility: Regeneron Study Site
state: Osaka
city: Suita
country: Japan
facility: Regeneron Study Site
state: Osaka
city: Amsterdam
country: Netherlands
facility: Clinical Trial Site
city: Johannesburg
country: South Africa
facility: Regeneron Study Site
state: Gauteng
city: Cape Town
country: South Africa
facility: Regeneron Study Site
state: Western Cape
city: Ivano-Frankivsk
country: Ukraine
facility: Clinical Trial Site
city: Kharkiv
country: Ukraine
facility: Clinical Trial Site #1
city: Kharkiv
country: Ukraine
facility: Clinical Trial Site #2
city: Kyiv
country: Ukraine
facility: Clinical Trial Site
city: Kyiv
country: Ukraine
facility: Rgeneron Study Site
Study officials:
affiliation: Regeneron Pharmaceuticals
name: Clinical Trial Management
role: STUDY_DIRECTOR
References: