In short
ZODASIRAN is being studied in clinical trials for people with homozygous familial hypercholesterolemia, a rare condition that causes very high LDL cholesterol. These trials are designed to evaluate how well it lowers LDL cholesterol and to compare it with placebo in adolescent and adult participants.
Key points
- ZODASIRAN is being studied in a Phase 3 trial for adolescents and adults with homozygous familial hypercholesterolemia (HoFH). The study is designed to compare ZODASIRAN with placebo. The main outcome is the percent change in fasting LDL cholesterol from the start of the study to Month 12. The trial is interventional, meaning researchers assign treatments and measure the results. A total of 61 participants are planned for enrollment. The trial status is listed as Authorised.
Trial overview
The provided trial information describes one Phase 3 study of ZODASIRAN called YOSEMITE. It is an interventional study, which means researchers give a study treatment and then measure the results.
This study is focused on people with homozygous familial hypercholesterolemia (HoFH), a rare inherited condition that causes very high LDL cholesterol. The brief study summary says the goal is to show a reduction in LDL cholesterol with ZODASIRAN compared with placebo.
Who can participate
The study is for adolescent and adult subjects with HoFH. The source data do not give the full list of entry rules, so the exact age limits and other requirements are not provided here.
Because the trial is for HoFH, it is aimed at people who already have this specific condition rather than the general population.
What is being measured
The main endpoint is the percent change from baseline to Month 12 in fasting LDL-C during the randomized period. An endpoint is the main result researchers use to judge whether a trial is working.
Baseline means the starting point before treatment begins, and fasting LDL-C means LDL cholesterol measured after not eating for a period of time. The study is set up to see whether ZODASIRAN lowers this value more than placebo over 12 months.
Trial design and treatment groups
The trial compares ZODASIRAN with a placebo, which is a look-alike treatment with no active study drug. The treatment listed in the source is “Drug: Placebo of ZODASIRAN,” showing that participants are compared against a placebo group.
The intervention listed for the active study treatment is “ARO-ANG3 (200 mg milligram(s), SUBCUTANEOUS INJECTION),” but the trial title and summary identify the study as a ZODASIRAN trial. The source data do not provide more details about how the treatment is given beyond the injection route shown in the intervention list.
Study status and size
The trial status is listed as Authorised, meaning the study has been approved to proceed in the source record. The planned enrollment is 61 participants.
This is a relatively small study population, which is common in trials for rare diseases like HoFH. The available data do not include final results, so the trial record here describes the study plan rather than the outcome.