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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 8 August 2022
Main ID:  EUCTR2019-001931-30-AT
Date of registration: 03/03/2020
Prospective Registration: Yes
Primary sponsor: Regeneron Pharmaceuticals, Inc.
Public title: A trial to evaluate the Efficacy and Safety of Evinacumab in Paediatric Patients With Homozygous Familial Hypercholesterolemia
Scientific title: A Three-Part, Single-Arm, Open-Label Study to Evaluate the Efficacy, Safety, and Pharmacokinetics of Evinacumab in Pediatric Patients with Homozygous Familial Hypercholesterolemia
Date of first enrolment: 17/08/2020
Target sample size: 20
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2019-001931-30
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no
Randomised: no
Open: yes
Single blind: no
Double blind: no
Parallel group: no
Cross over: no
Other: no
If controlled, specify comparator, Other Medicinial Product: no
Placebo: no
Other: no
 
Phase:  Human pharmacology (Phase I): yes Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no
Countries of recruitment
Australia Austria Netherlands Taiwan United States
Contacts
Name: Clinical Trial Information   
Address:  777 Old Saw Mill River Road 10591 Tarrytown, NY United States
Telephone:
Email: clinicaltrials@regeneron.com
Affiliation:  Regeneron Pharmaceuticals, Inc.
Name: Clinical Trial Information   
Address:  777 Old Saw Mill River Road 10591 Tarrytown, NY United States
Telephone:
Email: clinicaltrials@regeneron.com
Affiliation:  Regeneron Pharmaceuticals, Inc.
Key inclusion & exclusion criteria
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 include a daily statin.
5. Willing and able to comply with clinic visits and study-related procedures
6. 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)
Are the trial subjects under 18? yes
Number of subjects for this age range: 20
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion criteria:
1. 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
2. For patients entering Part A, unable to temporarily discontinue apheresis from the baseline visit through the week 4 visit
3. 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).
4. Plasmapheresis within 8 weeks of the screening visit, or plans to undergo plasmapheresis during Part A or Part B
5. Presence of any clinically significant uncontrolled endocrine disease known to influence serum lipids or lipoproteins
6. Newly diagnosed (within 3 months prior to randomization visit) diabetes mellitus or poorly controlled diabetes as defined in the protocol

Additional exclusion criteria apply


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes
Health Condition(s) or Problem(s) studied
Homozygous familial hypercholesterolemia
MedDRA version: 21.0 Level: LLT Classification code 10020604 Term: Hypercholesterolemia System Organ Class: 100000004861
Therapeutic area: Body processes [G] - Metabolic Phenomena [G03]
Intervention(s)

Product Name: Evinacumab
Product Code: REGN1500
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: Evinacumab
CAS Number: 1446419-85-7
Current Sponsor code: REGN1500
Other descriptive name: EVINACUMAB
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentration number: 150-

Primary Outcome(s)
Timepoint(s) of evaluation of this end point: 1. Up to week 24
2. Week 24
Main Objective: 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.
Secondary Objective: 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
Primary end point(s): 1. Pharmacokinetic (PK) parameters for evinacumab (Part A)
2. Percent change in calculated low-density lipoprotein cholesterol (LDL-C) from baseline to week 24 (part B)
Secondary Outcome(s)
Secondary end point(s): 1. Incidence of treatment-emergent adverse events (TEAE) and other safety variables over time (Part A & B)
2. Percent change in Apoliprotein (Apo) B from baseline to week 24 (Part B)
3. Percent change in non-High-density lipoprotein cholesterol (HDL-C) from baseline to week 24 (Part B)
4. Percent change in total cholesterol (TC) from baseline to week 24 (Part B)
5. Proportion of patients with =50% reduction in calculated LDL-C at week 24 (Part B)
6. Percent change in calculated LDL-C from baseline to week 24 in patients who have negative/negative and null/null mutations (Part B)
7. Percent change in lipoprotein a [Lp(a)] from baseline to week 24 (Part B)
8. The absolute change in LDL-C at week 24 (Part B)
9. Concentrations of total evinacumab over time (Part B)
10. PK parameters (Part B)
11. Incidence and titer of treatment-emergent anti-drug antibodies (ADA) over time (Part B)
Timepoint(s) of evaluation of this end point: 1. Up to week 68
2.-7. Week 24
8.-10. Up to week 68
Secondary ID(s)
R1500-CL-17100
Source(s) of Monetary Support
Regeneron Pharmaceuticals, Inc.
Secondary Sponsor(s)
Ethics review
Status: Approved
Approval date: 17/08/2020
Contact:
Results
Results available:
Date Posted:
Date Completed:
URL:
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