Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
Starting soon · Phase 3
Conditions studied: Homozygous Familial Hypercholesterolemia
In brief
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.
Key facts
- Study ID
- NCT07473843
- Run by
- Arrowhead Pharmaceuticals
- People needed
- 12
- Starts
- 2026-08-01
- Expected to finish
- 2028-08-01
- Last updated by the study team
- 2026-05-29
Who can join
Age: 12 and older, up to 17. Sex: any. Healthy volunteers: not accepted.
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)
- NOTE: Additional inclusion/exclusion criteria may apply per protocol
Full record on ClinicalTrials.gov
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