Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)

• Register: ClinicalTrials.gov
• Last refreshed on: 27 February 2024
• Main ID: NCT02624869
• Date of registration: 22/10/2015
• Prospective Registration: Yes
• Primary sponsor: Amgen
• Public title: Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia) HAUSER-OLE
• 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)
• Date of first enrolment: September 10, 2016
• Target sample size: 163
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT02624869
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 3

Countries of recruitment

Australia, Austria, Belgium, Brazil, Canada, Colombia, Czechia, Greece, Hungary, Italy, Malaysia, Netherlands, Norway, Poland, Portugal, Russian Federation, Slovenia, South Africa, Spain, Switzerland, Turkey, United Kingdom, United States

Contacts

• Name: MD
• Affiliation: Amgen

Key inclusion & exclusion criteria

Inclusion Criteria:

Heterozygous Familial Hypercholesterolemia (HeFH):

-Completed Study 20120123 (NCT02392559) while still on assigned investigational product and
did not experience a treatment-related serious adverse event

Homozygous Familial Hypercholesterolemia (HoFH):

- Male or female, = 10 to = 17 years of age at time of enrollment

- Diagnosis of HoFH

- On a low-fat diet and receiving background lipid-lowering therapy

- Lipid-lowering therapy unchanged for = 4 weeks prior to LDL-C screening; fibrates must
be stable for at least 6 weeks prior to screening.

- Fasting LDL-C at screening = 130 mg/dL (3.4 mmol/L)

- Fasting triglycerides = 400 mg/dL (4.5 mmol/L)

Exclusion Criteria:

-Currently receiving treatment in another investigational device or drug study, or less
than 30 days since ending treatment on another investigational device or drug study(s);
except Study 20120123

HoFH:

- Moderate to severe renal dysfunction

- Active liver disease or hepatic dysfunction,

- Creatine kinase > 3 times the upper limit of normal (ULN) at screening



Age minimum: 10 Years
Age maximum: 17 Years
Gender: All

Health Condition(s) or Problem(s) studied

Familial Hypercholesterolemia

Intervention(s)

Biological: Evolocumab

Primary Outcome(s)

Number of Participants With Treatment-emergent Adverse Events (TEAEs) [Time Frame: From first dose of evolocumab in this study up to and including 30 days after the last dose or up to the end of study date, whichever was earlier; up to 80 weeks.]

Secondary Outcome(s)

Change From Baseline in Height at Weeks 24, 48, and 80 [Time Frame: Baseline and weeks 24, 48, and 80]

Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT) [Time Frame: Baseline and week 80]

Change From Baseline in Weight at Weeks 24, 48, and 80 [Time Frame: Baseline and weeks 24, 48, and 80]

Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in LDL-C in HeFH Participants [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in LDL-C in HoFH Participants [Time Frame: Baseline and week 80]

Number of Participants With Liver Function Test Abnormalities at Week 80 [Time Frame: Week 80]

Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants [Time Frame: Baseline and week 80]

Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants [Time Frame: Baseline and week 80]

Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels [Time Frame: Baseline and week 80]

Number of Participants With Change in Tanner Staging From Baseline to Week 80 [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in Estradiol Levels [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in Cortisol Levels [Time Frame: Baseline and week 80]

Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants [Time Frame: Baseline and week 80]

Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 [Time Frame: Week 80]

Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants [Time Frame: Baseline and week 80]

Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants [Time Frame: Baseline and week 80]

Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants [Time Frame: Baseline and week 80]

Change From Baseline to Week 80 in Testosterone Levels [Time Frame: Baseline and week 80]

Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants [Time Frame: Baseline and week 80]

Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants [Time Frame: Baseline and week 80]

Secondary ID(s)

20120124

2015-002276-25

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 14/01/2022
• URL: https://clinicaltrials.gov/ct2/show/results/NCT02624869