Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia (NCT07473843) | Clinical Trial Compass
Not Yet RecruitingPhase 3
Study of Zodasiran in Adolescent Participants With Homozygous Familial Hypercholesterolemia
12 participantsStarted 2026-03
Plain-language 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.
Who can participate
Age range12 Years – 17 Years
SexALL
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AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
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
* 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
What they're measuring
1
Percent Change from Baseline to Month 12 in Fasting LDL-C