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RecruitingPhase 3

Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia

Arrowhead Pharmaceuticals · NCT07473843

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Opens the official ClinicalTrials.gov record, with the study team’s contacts. Bored Studies doesn’t enroll anyone.

Key facts

Compensation
Not posted. Ask the study team before screening.
Who can join
Ages 12–17 · All sexes
Healthy volunteers
Not accepted (a condition is required)
Study type
Interventional · Phase 3
Target enrollment
10
US sites recruiting
1
Study start
October 2026
Record last updated
October 26, 2026

Status re-checked against ClinicalTrials.gov within the last 12 hours.

What the study is about

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.

Being studied: Zodasiran

Conditions: Homozygous Familial Hypercholesterolemia

Eligibility criteria

Copied from the official record. The study team makes the final decision at screening.

Show full criteria

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

Recruiting US locations

Study contacts (from the official record)

  • Medical Monitor · 626-304-3400 · SPRUCE@arrowheadpharma.com
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Study of Zodasiran in Adolescent Participants With Homozygous… (NCT07473843) | Bored Studies