Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hyp… (NCT04233918) | Clinical Trial Compass
CompletedPhase 3
Evaluate the Efficacy and Safety of Evinacumab in Pediatric Patients With Homozygous Familial Hypercholesterolemia
United States20 participantsStarted 2020-06-29
Plain-language summary
The primary objective for Part A of the study is to assess the pharmacokinetics (PK) of evinacumab in pediatric patients with homozygous familial hypercholesterolemia (HoFH).
The primary objective for Part B of the study is to demonstrate a reduction of low-density lipoprotein cholesterol (LDL-C) by evinacumab in pediatric (5 to 11 years of age) patients with HoFH.
The secondary objective for Part A of the study is to evaluate the safety and tolerability of evinacumab administered intravenous (IV) in pediatric patients with HoFH.
The secondary objectives for Part B of the study are:
* To evaluate the effect of evinacumab on other lipid parameters (ie, apolipoprotein B (Apo B), non-high-density lipoprotein cholesterol (non-HDL-C), total cholesterol (TC), lipoprotein a \[Lp(a)\]) in pediatric patients with HoFH
* To evaluate the safety and tolerability of evinacumab administered IV in pediatric patients with HoFH
* To assess the PK of evinacumab in pediatric patients with HoFH
* To assess the immunogenicity of evinacumab in pediatric patients with HoFH over time
* To evaluate patient efficacy by mutation status
Who can participate
Age range5 Years – 11 Years
SexALL
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Inclusion criteria
✓. Diagnosis of functional HoFH by either genetic or clinical criteria as defined in the protocol
✓. LDL-C \>130 mg/dL at the screening visit
✓. Body weight ≥15 kg
✓. Receiving stable maximally tolerated therapy\*at the screening visit \*Maximally tolerated therapy could include a daily statin.
✓. Willing and able to comply with clinic visits and study-related procedures
✓. Parent(s) or legal guardian(s) must provide the signed informed consent form (ICF). Patients ≥5 years of age (or above age determined by the IRB/EC and in accordance with the local regulations and requirements) must also provide informed assent forms (IAFs) to enroll in the study, and sign and date a separate IAF or ICF signed by the parent(s)/legal guardian(s) (as appropriate based on local regulations and requirements)
Exclusion criteria
✕. Background pharmacologic LMT, nutraceuticals or over-the-counter (OTC) therapies known to affect lipids, at a dose/regimen that has not been stable for at least 4 weeks (8 weeks for PCSK9 inhibitors) before the screening visit and patient is unwilling to enter the run-in period
What they're measuring
1
Part A: Maximum Observed Serum Concentration (Cmax) of Evinacumab
Timeframe: At day 12
2
Part A: Area Under the Serum Concentration-Time Curve From Time Zero to the Time of the Last Measurable Concentration (AUClast) of Evinacumab
Timeframe: Up to Week 12
3
Part A: Terminal Half-Life (t1/2) of Evinacumab
Timeframe: Up to week 12
4
Part B: Percent Change in Calculated Low-Density Lipoprotein Cholesterol (LDL-C) From Baseline to Week 24
✕. For patients entering Part A, unable to temporarily discontinue apheresis from the baseline visit through the week 4 visit
✕. Receiving lipid apheresis, a setting (if applicable) and schedule that has not been stable for approximately 8 weeks before the screening visit or an apheresis schedule that is not anticipated to be stable over the duration of the treatment period (48 weeks).
✕. Plasmapheresis within 8 weeks of the screening visit, or plans to undergo plasmapheresis during Part A or Part B
✕. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
✕. Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or poorly controlled diabetes as defined in the protocol