New shot could help teens with rare, severe high cholesterol
NCT ID NCT07473843
First seen Jun 27, 2026 · Last updated Jun 27, 2026
Summary
This study tests a new drug called zodasiran in 12 teens aged 12 to 17 with homozygous familial hypercholesterolemia, a rare genetic condition causing very high cholesterol. The drug is given as a shot under the skin. The main goal is to see if it safely lowers bad cholesterol after 12 months. Participants must already be on other cholesterol-lowering treatments.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 3
Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Aug 2026
An estimate. Start dates often move.
- Expected to finish
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Aug 2028
An estimate. End dates often move.
- Lead sponsor
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A company
The lead sponsor is a pharmaceutical, biotech, or medical-device company.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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12 to 17 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
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
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.
More trials for these conditions
Other studies related to the condition(s) this trial covers.
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- New repository aims to unlock secrets of rare, deadly cholesterol disease
- Worldwide effort to unlock secrets of rare cholesterol disease
- New drug shows promise for rare High-Cholesterol disease
- Experimental drug targets stubborn cholesterol in rare disease