Ireland
--:--IST
Recruiting Phase 3

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

NCT07473843 · tracked via the Priya Life Science Denmark tracker
Phase
Phase 3
Started
2026-10
Last updated
2026-10-06

Condition(s) studied

Homozygous Familial Hypercholesterolemia

Investigational drug(s) / intervention(s)

Zodasiran →

Zodasiran: By sc injection

Study summary

This study will evaluate the efficacy and safety of zodasiran subcutaneous (sc) injection in participants 12 to \<18 years of age with genetically or clinically diagnosed homozygous familial hypercholesterolemia (HoFH) and low-density lipoprotein cholesterol (LDL-C) ≥116 milligrams per deciliter (mg/dL) on maximally tolerated lipid-lowering therapy.

Eligibility

Sex
ALL
Min age
12 Years
Max age
17 Years
Healthy volunteers
No
Inclusion Criteria: * Adolescents 12 to \<18 years of age who are nonpregnant, nonlactating, and do not plan to become pregnant during the study * Body weight ≥35 kilograms (kg) at screening * HoFH based on a supportive genetic test (from a source-verifiable medical record or based on screening genotype) or clinical diagnosis * Screening LDL-C ≥116 mg/dL (3 mmol/L) * Screening hemoglobin A1c (HbA1c) ≤9.5% * Total bilirubin \<2×upper limit of normal (ULN), unless in previously confirmed cases of Gilbert's syndrome * Alanine aminotransferase or aspartate aminotransferase \<3×ULN Exclusion Criteria: * 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 * Use of evinacumab within 3 months before Day 1 * Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen) NOTE: Additional inclusion/exclusion criteria may apply per protocol

Primary outcome measure(s)

  • Percent Change from Baseline to Month 12 in Fasting LDL-C — Baseline, Month 12

Trial sites (2)

FacilityCityRegionStatus
Research Site St Louis Missouri Recruiting
Research Site Viborg Denmark Recruiting
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT07473843 on ClinicalTrials.gov ↗ ← All trials in Denmark