Phase II Clinical Trial to Evaluate the Efficacy and Safety of SYH2070 Injection in Participants … (NCT07591298) | Clinical Trial Compass
Not Yet RecruitingPhase 2
Phase II Clinical Trial to Evaluate the Efficacy and Safety of SYH2070 Injection in Participants With Homozygous Familial Hypercholesterolemia
18 participantsStarted 2026-05-24
Plain-language summary
This trial is a multicenter, open-label, phase II clinical study, aiming to evaluate the efficacy and safety of SYH2070 injection in participants with HoFH.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion criteria
. Age ≥18 years old, male or female, and weight ≥40 kg.
. Genetic diagnosis or clinical diagnosis of HoFH.
. Receiving stable and tolerable lipid-lowering treatment or other drugs for chronic disease treatment for certain periods before the study, and maintaining the stable -treatments throughout the study.
. Fasting serum LDL-C ≥2.6 mmol/L.
. Fasting TG during screening is ≤5.6 mmol/L
. BMI\< 40 kg/m ² during screening
. Understand the study procedures, voluntarily participate, and sign the informed consent form.
Exclusion criteria
. The genetic diagnosis was for heterozygous familial hypercholesterolemia;
. During the screening process, the participant has uncontrolled other diseases that affect blood lipids or lipoproteins (such as nephrotic syndrome, severe liver diseases, glycogen storage disease, systemic lupus erythematosus, Cushing's syndrome, etc.) that the researchers believed would interfere with the accurate assessment of the study validity;
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The percentage change in serum LDL-C level from baseline
. The participant has received or is receiving monoclonal antibodies targeting ANGPTL3 within 5 months or 5 half-lives (whichever is longer) before the LDL-C test during the screening period;
. Within 12 months before the LDL-C test during the screening period, use of any ASO or siRNA type drugs;
. Those who used any of the following treatments within the specified time limit before the LDL-C test: 1) mipomersen (within 5 months); 2) bepatide acid (within 4 weeks); 3) lipid purification or plasma exchange (within 8 weeks); 4) liver transplantation or CRISPR-based gene editing treatment (at any time);
. During the screening period, within 5 months or 5 half-lives (whichever is longer) prior to the LDL-C test, the subject had received significant medications other than lipid-lowering drugs that affected LDL-C levels (such as oral or intravenous glucocorticoids, tacrolimus, cyclosporine, sirolimus, estrogens, vitamin A derivatives, antidepressants, etc.);
. At the time of screening, the subject was using medications for thyroid disorders and the use of thyroid disorder medications was stable for less than 12 weeks before the LDL-C test;
. History of allergic or suspected allergic reactions to oligonucleotide drugs or excipients of investigational drugs;