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A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE)

60 patients around the world

Available in United States, Brazil

Arrowhead Pharmaceuticals

60Patients around the world

This study is for people with

Hypercholesterolemia

Familial hypercholesterolemia

From 12 Years

All Gender

: Age ≥12 years, non pregnant, non lactating, do not plan to become pregnant during the study.
Body weight ≥35 kg at Screening as patients could theoretically be <35 kg as the study continues.
HoFH based on a supportive genetic test or a clinical diagnosis (total cholesterol >500 mg/dL[13 mmol/L] OR treated LDL-C concentration of ≥300 mg/dL [≥8 mmol/L] either accompanied by TGs <300 mg/dL [3.4 mmol/L] AND both parents with documented total cholesterol >250 mg/dL [6.5 mmol/L] OR cutaneous or tendinous xanthoma before 10 years of age).
LDL-C ≥70 mg/dL (1.8 mmol/L). For adolescents 12 to <18 years of age, screening LDL-C ≥116 mg/dL (3 mmol/L).
Hemoglobin A1c (HbA1c) ≤9.5%.
Total bilirubin <2xULN, unless in previously confirmed cases of Gilbert's syndrome.
Alanine aminotransferase or aspartate aminotransferase <3×ULN.
On standard of care, maximally tolerated lipid-lowering therapy to include a maximally tolerated statin, ezetimibe, and a PCSK9 inhibitor.

: Use of a hepatocyte-targeted siRNA within 365 days before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks).
Use of an antisense oligonucleotide molecule within 3 months before Day 1 (except inclisiran, which is permitted; administration of inclisiran and study drug must be separated by at least 4 weeks).
Use of evinacumab within 3 months before Day 1. Evinacumab use is prohibited during the study.
Non-response to evinacumab, defined as LDL-C reduction <15% from baseline after 2 doses.
Use of any other investigational agent or device within 30 days or 5 half-lives (whichever is longer) before Day 1.
Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen).
Estimated glomerular filtration rate <30 mL/min.

SponsorArrowhead Pharmaceuticals

Study typeInterventional

Conditions

Familial hypercholesterolemia

Requirements

From 12 YearsAll Gender

Unique study IDclinicaltrials.gov

NCT07037771