Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT04233918 | Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia | PHASE3 | COMPLETED | 20 | — | — | Jun 29, 2020 | May 30, 2023 | Jul 16, 2024 | 11 | United States, Australia +4 |
| NCT03409744 | Evaluate the Long-Term Safety and Efficacy of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | PHASE3 | COMPLETED | 116 | — | — | Mar 13, 2018 | Apr 13, 2023 | Apr 8, 2025 | 38 | United States, Australia +10 |
| NCT03399786 | Efficacy and Safety of Evinacumab in Patients With Homozygous Familial Hypercholesterolemia | PHASE3 | COMPLETED | 65 | — | — | Jan 18, 2018 | Mar 17, 2020 | May 18, 2021 | 30 | United States, Australia +9 |
Cmax was obtained directly from the plasma concentration versus time curve.
AUClast was defined as area under the serum concentration time-curve from zero to the last measured concentration.
T1/2 was defined as the time required for the plasma concentration of drug to decrease 50 percent in the final stage of its elimination.
Percent change was calculated as 100 multiplied by (calculated LDL-C value at Week 24 minus calculated LDL-C value at baseline) divided by calculated LDL-C value at baseline.
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.
Percent change was calculated as 100x(calculated LDL-C value at Week 24 - calculated LDL-C value at baseline)/calculated LDL-C value at baseline. The baseline LDL-C value was the last calculated LDL-C value obtained before the first dose of double-blind-study drug. The calculated LDL-C at week 24 was the LDL-C value obtained within the week 24 efficacy analysis window, regardless of adherence to treatment and subsequent therapies (intent-to-treat \[ITT\] estimand). The ITT population included all randomized participants who received at least one dose or part of a dose of double-blind study drug. Participants in the ITT population were analyzed according to the treatment group allocated by randomization (i.e., as randomized participant group).
| Arm | Type | Description |
|---|---|---|
| Evinacumab | EXPERIMENTAL | Part A: Single intravenous (IV) dose Part B: IV dose every 4 weeks (Q4W) until week 20 Part C: IV dose Q4W |
| Placebo | EXPERIMENTAL | - |
| Name | Type | Description |
|---|---|---|
| Evinacumab | DRUG | Part A: Single IV dose Part B \& C: IV dose Q4W |
| Placebo | DRUG | IV administration of placebo |
Key Inclusion Criteria: 1. Diagnosis of functional HoFH by either genetic or clinical criteria as defined in the protocol 2. LDL-C \>130 mg/dL at the screening visit 3. Body weight ≥15 kg 4. Receiving stable maximally tolerated therapy\*at the screening visit \*Maximally tolerated therapy could inc...