Phase 3
Completed N=163
Safety, Tolerability and Efficacy of Evolocumab (AMG 145) in Children With Inherited Elevated Low-density Lipoprotein Cholesterol (Familial Hypercholesterolemia)
Source: ClinicalTrials.gov NCT02624869 ↗Enrolled (actual)
163
Serious AEs
3.7%
Results posted
Jan 2022
Primary outcomePrimary: Number of Participants With Treatment-emergent Adverse Events (TEAEs) — 36; 69; 7; 25 Participants
◆ Published Evidence
Established
48citations · ~12 / year
Paediatric patients with heterozygous familial hypercholesterolaemia treated with evolocumab for 80 weeks (HAUSER-OLE): a single-arm, multicentre, open-label extension of HAUSER-RCT.
Summary
The main purpose of this study is to describe the safety and tolerability of 80 weeks of subcutaneous (SC) evolocumab when added to standard of care in children 10 to 17 years of age with familial hypercholesterolemia.
Linked Publications (3)
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Paediatric patients with heterozygous familial hypercholesterolaemia treated with evolocumab for 80 weeks (HAUSER-OLE): a single-arm, multicentre, open-label extension of HAUSER-RCT.
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Evolocumab Treatment in Pediatric Patients With Homozygous Familial Hypercholesterolemia: Pooled Data From Three Open-Label Studies.
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Evolocumab in paediatric heterozygous familial hypercholesterolaemia: cognitive function during 80 weeks of open-label extension treatment.
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-emergent Adverse Events (TEAEs) |
36; 69; 7; 25; 56; 5 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HeFH Participants |
-36.01; -34.96 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Low-density Lipoprotein Cholesterol (LDL-C) in HoFH Participants |
-14.29 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Non-HDL-C in HeFH Participants |
-32.37; -31.95 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Non-HDL-C in HoFH Participants |
-13.03 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Apolipoprotein B in HeFH Participants |
-27.10; -24.15 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Apolipoprotein B in HoFH Participants |
-19.17 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HeFH Participants |
-28.78; -28.32 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Total Cholesterol/HDL-C Ratio in HoFH Participants |
3.71 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Apolipoprotein B / Apolipoprotein A1 Ratio in HeFH Participants |
-31.00; -29.89 | — |
| SECONDARY Percent Change From Baseline to Week 80 in Apolipoprotein B/Apolipoprotein A1 Ratio in HoFH Participants |
-2.96 | — |
| SECONDARY Change From Baseline to Week 80 in LDL-C in HeFH Participants |
-67.2; -63.1 | — |
| SECONDARY Change From Baseline to Week 80 in LDL-C in HoFH Participants |
-36.5 | — |
| SECONDARY Change From Baseline to Week 80 in Estradiol Levels |
131.3; 48.2; 283.0 | — |
| SECONDARY Change From Baseline to Week 80 in Testosterone Levels |
5.282; 3.230; 2.916 | — |
| SECONDARY Change From Baseline to Week 80 in Follicle Stimulating Hormone (FSH) Levels |
1.88; 0.60; 1.18 | — |
| SECONDARY Change From Baseline to Week 80 in Luteinizing Hormone (LH) Levels |
2.88; 1.04; 1.76 | — |
| SECONDARY Change From Baseline to Week 80 in Adenocorticotropic Hormone (ACTH) Levels |
0.78; 0.55; -0.75 | — |
| SECONDARY Change From Baseline to Week 80 in Dehydroepiandrosterone Sulfate (DHEA-S) Levels |
1.051; 0.956; 0.944 | — |
| SECONDARY Change From Baseline to Week 80 in Cortisol Levels |
29.81; 51.18; 57.26 | — |
| SECONDARY Number of Participants With Liver Function Test Abnormalities at Week 80 |
0; 0; 0; 0; 0; 0 | — |
| SECONDARY Number of Participants With Abnormalities in Levels of Creatine Kinase (CK) at Week 80 |
0; 0; 1; 0; 0; 0 | — |
| SECONDARY Change From Baseline to Week 80 in Carotid Intima-media Thickness (cIMT) |
-0.019; -0.012; 0.006 | — |
| SECONDARY Change From Baseline in Height at Weeks 24, 48, and 80 |
158.1; 157.9; 149.4; 2.8; 2.0; 1.6 | — |
| SECONDARY Change From Baseline in Weight at Weeks 24, 48, and 80 |
52.8; 57.0; 42.7; 3.3; 2.3; 3.4 | — |
| SECONDARY Number of Participants With Change in Tanner Staging From Baseline to Week 80 |
13; 20; 6; 14; 21; 6 | — |
Eligibility 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
Data sourced from ClinicalTrials.gov (NCT02624869) and the linked publication. Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication. Informational only — not medical advice.