Phase 3
N=116
Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia
Homozygous Familial Hypercholesterolemia
Bottom Line
View on ClinicalTrials.gov: NCT03409744 ↗Enrolled (actual)
116
Serious AEs
23.3%
Results posted
Apr 2024
Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216 — 93; 27 Number of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 3
- Interventions
- evinacumab (Drug)
- Age
- Pediatric, Adult, Older Adult · 12+ yrs
- Sex
- All
- Sponsor
- Regeneron Pharmaceuticals
- Primary completion
- Apr 2023
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) Up to Week 216 |
93; 27 | — |
| SECONDARY Percent Change in Low-Density Lipoprotein Cholesterol (LDL-C) Over Time |
-46.45; -43.64; -43.88; -45.17; -38.00; -33.10 | — |
| SECONDARY Absolute Change in LDL-C Over Time |
-142.1; -132.0; -132.8; -135.1; -131.4; -132.5 | — |
| SECONDARY Percent Change in Apolipoprotein B (Apo B) Over Time |
-39.74; -36.98; -35.85; -37.60; -32.54; -27.73 | — |
| SECONDARY Absolute Change in Apo B Over Time |
-78.0; -70.5; -69.4; -73.9; -71.5; -70.7 | — |
| SECONDARY Percent Change in Non-High-Density Lipoprotein Cholesterol (HDL-C) Over Time |
-48.47; -46.14; -45.15; -46.69; -40.88; -35.85 | — |
| SECONDARY Absolute Change in Non-HDL-C Over Time |
-153.7; -143.9; -144.1; -147.3; -144.5; -142.9 | — |
| SECONDARY Percent Change in Total Cholesterol (TC) Over Time |
-47.04; -44.17; -43.78; -44.58; -40.33; -36.92 | — |
| SECONDARY Absolute Change in TC Over Time |
-167.3; -157.4; -158.2; -160.7; -157.2; -153.8 | — |
| SECONDARY Percent Change in Triglycerides (TGs) Over Time |
-45.97; -46.93; -43.25; -46.50; -48.28; -42.06 | — |
| SECONDARY Absolute Change in TGs Over Time |
-64.4; -68.1; -66.7; -68.8; -75.2; -66.7 | — |
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
- 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
Data sourced from ClinicalTrials.gov (NCT03409744). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.