Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia

• Register: EUCTR
• Last refreshed on: 5 July 2021
• Main ID: EUCTR2015-002276-25-HU
• Date of registration: 12/04/2016
• Prospective Registration: Yes
• Primary sponsor: Amgen Inc
• Public title: Study to assess the safety and efficacy of evolocumab in children aged 10 to 17 years with Heterozygous Familial Hypercholesterolemia or Homozygous Familial Hypercholesterolemia
• Scientific title: Open-label, Single-arm, Multicenter Study to Evaluate the Safety, Tolerability and Efficacy of Evolocumab for LDL-C Reduction, as Add-on to Diet and Lipid-lowering Therapy, in Pediatric Subjects From 10 to 17 Years of Age With Heterozygous Familial Hypercholesterolemia (HeFH) or Homozygous Familial Hypercholesterolemia (HoFH) - HAUSER-OLE
• Date of first enrolment: 06/06/2016
• Target sample size: 115
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2015-002276-25
• 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): no Therapeutic exploratory (Phase II): no Therapeutic confirmatory - (Phase III): yes Therapeutic use (Phase IV): no

Countries of recruitment

Australia, Austria, Belgium, Brazil, Canada, Colombia, Czech Republic, Germany, Greece, Hungary, Netherlands, New Zealand, Norway, Poland, Portugal, Slovenia, South Africa, Spain, Turkey, United Kingdom

Contacts

• Name: IHQ Medical Info - Clinical Trials
• Affiliation: Amgen (EUROPE) GmbH

• Name: IHQ Medical Info - Clinical Trials
• Affiliation: Amgen (EUROPE) GmbH

Key inclusion & exclusion criteria

Inclusion criteria:
Subjects with HeFH:
-Completed Study 20120123 while still on assigned investigational product.
Subjects with HoFH:
-Male or female, = 10 to = 17 years of age at time of enrollment
-Diagnosis of HoFH by genetic confirmation or a clinical diagnosis based on a
history of an untreated LDL cholesterol concentration > 500 mg/dL (13 mmol/L)
together with either xanthoma before 10 years of age or evidence of
heterozygous familial hypercholesterolemia in both parents.
All subjects:
- Subject must be on a low-fat diet and receiving background lipid-lowering therapy
-Lipid-lowering therapy, including statin dose, must be unchanged for = 4 weeks
prior to LDL-C screening; fibrates must be stable for at least 6 weeks prior to
screening.
Are the trial subjects under 18? yes
Number of subjects for this age range: 115
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:
Subjects with HoFH:
- estimated glomerular filtration rate (eGFR) < 30 ml/min/1.73m2; CK > 3x ULN; AST or ALT > 3x ULN; (all screening by central laboratory);
- known active infection or major hematologic, renal, metabolic, gastrointestinal or endocrine dysfunction;
- subject has taken a cholesterylester transfer protein (CETP) inhibitor in the last 12 months, or mipomersen or lomitapide in the last 5 months prior to LDL-C screening, or has received any therapy to inhibit proprotein convertase subtilisin/kexin type 9 (PCSK9) within 12 weeks prior to screening;
- subject has a history or evidence of any other clinically significant disorder, condition or disease, or planned or expected procedure that, in the opinion of the Investigator or Amgen physician, would pose a risk to subject safety or interfere with the study evaluation, procedures or completion.
The following are major exclusion criteria for all subjects:
- subjects cannot be receiving treatment in another investigational device or drug study, or less than 30 days since ending treatment on another investigational device or drug study;
- female subjects of childbearing potential cannot be pregnant or breast feeding or planning to become pregnant or planning to breast feed and must be willing to use acceptable method(s) of effective birth control (may include true sexual abstinence) during treatment with evolocumab and for an additional 15 weeks after the end of treatment with evolocumab.


Age minimum:
Age maximum:
Gender:
Female: yes
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Nutritional and Metabolic Diseases [C18]

Hypercholesterolemia
MedDRA version: 19.0 Level: LLT Classification code 10057100 Term: Homozygous familial hypercholesterolaemia System Organ Class: 100000004850
MedDRA version: 19.0 Level: LLT Classification code 10057099 Term: Heterozygous familial hypercholesterolaemia System Organ Class: 100000004850

Intervention(s)

Trade Name: Repatha 140 mg Solution for Injection in Pre-filled Pen
Product Name: Evolocumab
Product Code: AMG 145
Pharmaceutical Form: Solution for injection in pre-filled pen
INN or Proposed INN: EVOLOCUMAB
Current Sponsor code: AMG 145
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 140-
Pharmaceutical form of the placebo: Solution for injection in pre-filled pen
Route of administration of the placebo: Subcutaneous use

Primary Outcome(s)

Timepoint(s) of evaluation of this end point: Screening, day 1, weeks 4, 12, 24, 36, 48, 60, 72 and 80.

Secondary Objective: To describe percent change and change from baseline in LDL-C, and on percent
change from baseline in non-high-density lipoprotein cholesterol (non-HDL-C),
apolipoprotein B (ApoB), total cholesterol/HDL-C ratio, and ApoB/Apolipoprotein A-1
(ApoA1) ratio.

Main Objective: To describe the safety and tolerability of 80 weeks of SC evolocumab when added to
standard of care in pediatric subjects 10 to 17 years of age with HeFH or HoFH.

Primary end point(s): Subject incidence of treatment emergent adverse events

Secondary Outcome(s)

Timepoint(s) of evaluation of this end point: Baseline and week 80

Secondary end point(s): Percent change from baseline at week 80 in:
- LDL-C
- Non-HDL-C
- ApoB
- Total cholesterol/HDL-C ratio
- ApoB/ApoA1 ratio
• Change from baseline in LDL-C at week 80

Secondary ID(s)

20120124

2015-002276-25-DE

Source(s) of Monetary Support

Amgen Inc.

Ethics review

Status: Approved
Approval date: 30/05/2016
Contact: