Recruiting
Phase 3

Zodasiran

Sponsor:

Arrowhead Pharmaceuticals

Code:

NCT07473843

Conditions

Homozygous Familial Hypercholesterolemia

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

Interventions

Zodasiran

Study Details

Brief 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.

Conditions

Homozygous Familial Hypercholesterolemia

Study ID

NCT07473843

Start date

Oct, 2026

Status verified date

Oct, 2026

Completion date

Aug, 2028

Anticipated

Primary completion date

Jul, 2028

Anticipated

Eligibility Criteria

Eligibility Criteria

Sex: All

Age: 12 - 17

Healthy Volunteers: Not accepted

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

Study Design

Enrollment

10 participants

Anticipated

Intervention Model

Single group

Primary purpose

Treatment

Interventions and Outcome Measures

Arms

experimental: Zodasiran

Interventions

Zodasiran

By sc injection

Primary outcome measure

  • Percent Change from Baseline to Month 12 in Fasting LDL-C [ Time Frame: Baseline, Month 12 ]

Central Contacts and Locations

Central contacts

Medical Monitor

626-304-3400SPRUCE@arrowheadpharma.com

Locations

Research Site

Recruiting

St Louis, Missouri, United States, 63110

More Information

Sponsor

Arrowhead Pharmaceuticals

Last update posted

Oct 6, 2026

Last verified

Oct, 2026

Keywords

  • Hyperlipoproteinemia Type II
  • Lipid Metabolism, Inborn Errors
  • Metabolism, Inborn Errors
  • Genetic Diseases, Inborn
  • Congenital, Hereditary, and Neonatal Diseases and Abnormalities
  • Hyperlipoproteinemias
  • Hyperlipidemias
  • Dyslipidemias
  • Lipid Metabolism Disorders
  • Metabolic Diseases
  • Nutritional and Metabolic Diseases
  • Homozygous Familial Hypercholesterolemia
  • Zodasiran

Trial information was received from ClinicalTrials.gov and was last updated on 2026-10-07. This information was provided to ClinicalTrials.gov by Arrowhead Pharmaceuticals on 2026-10-06. Recruitment status is synced daily from ClinicalTrials.gov and may not reflect the sponsor's current status. Confirm during your call.