Efficacy, safety, and tolerability of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia: Rationale and design of the HAUSER-RCT.

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Efficacy, safety, and tolerability of evolocumab in pediatric patients with heterozygous familial hypercholesterolemia: Rationale and design of the HAUSER-RCT study Daniel Gaudet, MD, PhD, Gisle Langslet, MD, Samuel S. Gidding, MD, Ilse K. Luirink, MD, Andrea Ruzza, MD, PhD, Christopher Kurtz, MD, Chen Lu, PhD, Ransi Somaratne, MD, Frederick J. Raal, MBBCh, PhD, Albert Wiegman, MD, PhD Journal of Clinical Lipidology Volume 12, Issue 5, Pages 1199-1207 (September 2018) DOI: 10.1016/j.jacl.2018.05.007 Copyright © 2018 National Lipid Association Terms and Conditions

Figure 1 HAUSER-RCT study sites. Countries that house study sites are depicted in the map. The legend shows the number and percentage of study sites by region. Journal of Clinical Lipidology 2018 12, 1199-1207DOI: (10.1016/j.jacl.2018.05.007) Copyright © 2018 National Lipid Association Terms and Conditions

Figure 2 Study design and treatment schema. Patients considered for enrollment undergo screening assessments, including laboratory screening by a central laboratory. Approximately 150 eligible patients are randomized in a 2:1 ratio to receive 24 weeks of QM evolocumab or placebo. Randomization is stratified by screening low-density lipoprotein cholesterol (<4.1 mmol/L [160 mg/dL] vs ≥ 4.1 mmol/L) and age (<14 years vs ≥ 14 years). D, day; EOS, end of study; IP, investigational product; QM, once monthly; SC, subcutaneous; W, week. Journal of Clinical Lipidology 2018 12, 1199-1207DOI: (10.1016/j.jacl.2018.05.007) Copyright © 2018 National Lipid Association Terms and Conditions