NCT07473843

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.

Trial Health

65
Monitor

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
12

participants targeted

Target at below P25 for phase_3

Timeline
36mo left

Started Mar 2026

Typical duration for phase_3

Status
not yet recruiting

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress7%
Mar 2026May 2029

Study Start

First participant enrolled

March 1, 2026

Completed
10 days until next milestone

First Submitted

Initial submission to the registry

March 11, 2026

Completed
5 days until next milestone

First Posted

Study publicly available on registry

March 16, 2026

Completed
2.3 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

July 1, 2028

Expected
10 months until next milestone

Study Completion

Last participant's last visit for all outcomes

May 1, 2029

Last Updated

March 20, 2026

Status Verified

March 1, 2026

Enrollment Period

2.3 years

First QC Date

March 11, 2026

Last Update Submit

March 19, 2026

Conditions

Homozygous Familial Hypercholesterolemia

Keywords

Hyperlipoproteinemia Type IILipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic DiseasesHomozygous Familial HypercholesterolemiaZodasiran

Outcome Measures

Primary Outcomes (1)

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

    Baseline, Month 12

Secondary Outcomes (20)

  • Percent Change from Baseline to Month 12 in Fasting Apolipoprotein B (ApoB)

    Baseline, Month 12

  • Change from Baseline to Month 12 in Fasting ApoB

    Baseline, Month 12

  • Percent Change from Baseline to Month 12 in Fasting Non-High Density Lipoprotein Cholesterol (non-HDL-C)

    Baseline, Month 12

  • Change from Baseline to Month 12 in Fasting Non-HDL-C

    Baseline, Month 12

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

    Baseline, Month 12

  • +15 more secondary outcomes

Study Arms (1)

Zodasiran

EXPERIMENTAL
Drug: Zodasiran

Interventions

ZodasiranDRUG

By sc injection

Also known as: ARO-ANG3
Zodasiran

Eligibility Criteria

Age12 Years - 17 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17)

You may qualify if:

  • 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

You may not qualify if:

  • 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
  • Non-response to evinacumab, defined as LDL-C reduction \<15% from baseline after 2 doses
  • Use of systemic corticosteroids (unless used as replacement therapy for pituitary/adrenal disease with a stable regimen)

Contact the study team to confirm eligibility.

Sponsors & Collaborators

MeSH Terms

Conditions

Homozygous Familial HypercholesterolemiaHyperlipoproteinemia Type IILipid Metabolism, Inborn ErrorsMetabolism, Inborn ErrorsGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesHyperlipoproteinemiasHyperlipidemiasDyslipidemiasLipid Metabolism DisordersMetabolic DiseasesNutritional and Metabolic Diseases

Interventions

zodasiran

Central Study Contacts

Medical Monitor

CONTACT

626-304-3400SPRUCE@arrowheadpharma.com

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
INDUSTRY
Responsible Party
SPONSOR

Study Record Dates

First Submitted

March 11, 2026

First Posted

March 16, 2026

Study Start

March 1, 2026

Primary Completion (Estimated)

July 1, 2028

Study Completion (Estimated)

May 1, 2029

Last Updated

March 20, 2026

Record last verified: 2026-03

Data Sharing

IPD Sharing
Will not share