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

Regeneron Pharmaceuticals

Status and phase: Completed; Phase 3

Conditions: Homozygous Familial Hypercholesterolemia

Treatments: Drug: evinacumab

Study type: Interventional

Funder types: Industry

Identifiers

NCT03409744

R1500-CL-1719

2017-003170-13 (EudraCT Number)

Details and patient eligibility

About

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

Enrollment: 116 patients

Sex: All

Ages: 12+ years old

Volunteers: No Healthy Volunteers

Inclusion and exclusion 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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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116 participants in 1 patient group

evinacumab

Experimental group

Treatment:

Drug: evinacumab