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New treatment option for Familial Hypercholesterolemia
Official title Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)
ClinicalTrials.gov ID: NCT06458010
What this study is testing
What is YOLT-101?
YOLT-101 is an investigational medicine, given as an infusion into a vein, being studied as a potential treatment for familial hypercholesterolemia.
Plain-language explanation of the investigational treatment - it is being studied and is not an approved or proven therapy. The study team can confirm the details.
- What it's testing
- This study is a single arm, open, single dose escalation trial aimed at evaluating the safety and tolerability of YOLT-101 administration in patients with familial hypercholesterolemia; Determination of YOLT-101 OBD; Preliminary evaluation of the effects of single administration of YOLT-101 on plasma lipid and lipoprotein levels. Note: OBD is defined as the dosage at which plasma PCSK9 protein levels decrease between 60% and 95% from baseline on the 28th day after YOLT-101 administration.
- Phase 1: an early, usually small safety study
A plain-language read of the study's public ClinicalTrials.gov listing. The study team confirms the details.
Who can take part
Ages 18 to 75
You may be able to join if
- Male or female, aged 18 to 75 years inclusive, at the time of signing informed consent.
- Meets the diagnostic criteria for familial hypercholesterolemia.
- At screening, there is a mutation in the PCSK9 and/or ApoB and/or LDLR gene.
- At screening, weight is ≥40kg, and Body Mass Index (BMI) is \>18kg/m\^2.
- At screening, people must meet the following laboratory criteria: 5.1 Hematology: Absolute Neutrophil Count (ANC) ≥1.5×10\^9/L, Platelet (PLT)...
You likely can't join if
- Within at least 14 days (or 5 half-lives of the drug, whichever is longer, for small molecule/small nucleic acid drugs) or 2 months (for biological...
- Patients with poorly controlled hypertension (systolic blood pressure (SBP) ≥160mmHg and/or diastolic blood pressure (DBP) ≥100mmHg).
- Patients with poorly controlled diabetes (glycated hemoglobin \>8.5%).
- Those allergic to drugs or components of LNP-mRNA vaccines contained in lipid nanoparticles (LNP), or those who have experienced adverse reactions...
- Within 3 months before screening, those who smoke more than 5 cigarettes per day or consume an equivalent amount of nicotine or nicotine replacement...
- Within 3 months before screening, those with a history of alcohol abuse [consuming more than 14 units of alcohol per week (1 unit ≈ 360mL beer or...
See the full eligibility criteria
- Male or female, aged 18 to 75 years inclusive, at the time of signing informed consent.
- Meets the diagnostic criteria for familial hypercholesterolemia.
- At screening, there is a mutation in the PCSK9 and/or ApoB and/or LDLR gene.
- At screening, weight is ≥40kg, and Body Mass Index (BMI) is \>18kg/m\^2.
- At screening, people must meet the following laboratory criteria: 5.1 Hematology: Absolute Neutrophil Count (ANC) ≥1.5×10\^9/L, Platelet (PLT) ≥100×10\^9/L, Hemoglobin (HGB) ≥90 g/dL; 5.2 Liver Function: Aspartate...
- On moderate intensity or higher statin therapy (statin treatment stable for 4 weeks) with LDL-C ≥2.6mmol/L; for those with evidence of atherosclerosis, LDL-C ≥1.8mmol/L. (Evidence of atherosclerosis includes: 1. History...
- people and their partners must use effective contraceptive measures during the study period and for at least 6 months after the end of the main study.
- Voluntarily sign informed consent.
- Within at least 14 days (or 5 half-lives of the drug, whichever is longer, for small molecule/small nucleic acid drugs) or 2 months (for biological agents such as PCSK9 inhibitors) or 1 year (for PCSK9 small nucleic...
- Patients with poorly controlled hypertension (systolic blood pressure (SBP) ≥160mmHg and/or diastolic blood pressure (DBP) ≥100mmHg).
- Patients with poorly controlled diabetes (glycated hemoglobin \>8.5%).
- Those allergic to drugs or components of LNP-mRNA vaccines contained in lipid nanoparticles (LNP), or those who have experienced adverse reactions due to LNP-based drug treatment, such as: 4.1 After receiving LNP-based...
- Within 3 months before screening, those who smoke more than 5 cigarettes per day or consume an equivalent amount of nicotine or nicotine replacement products.
- Within 3 months before screening, those with a history of alcohol abuse [consuming more than 14 units of alcohol per week (1 unit ≈ 360mL beer or 45mL of liquor with 40% alcohol or 150mL of wine)]; or those who test...
- At screening, those with New York Heart Association (NYHA) defined class III-IV heart failure, or left ventricular ejection fraction \ 470ms, males \>450ms).
- Within 3 months before screening, those with poorly controlled severe arrhythmias, such as recurrent and highly symptomatic ventricular tachycardia, rapid ventricular response atrial fibrillation, or supraventricular...
- Within 3 months before screening, those with myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass grafting, severe deep vein thrombosis, or pulmonary embolism; those who...
- Those with diseases that significantly affect lipid levels and are uncontrollable, such as nephrotic syndrome, severe liver disease, Cushing's syndrome, thyroid dysfunction, etc. (those with hypothyroidism before...
- Those who have donated more than 500 mL of blood within 3 months before screening.
- Those who cannot or are unwilling to accept the required medication treatment plan before treatment.
- Those who have undergone antithrombotic treatment (such as warfarin, dabigatran, apixaban) within 14 days before enrollment.
- Those with a history of easy bleeding or coagulation disorders (such as liver cirrhosis, malignant hematological diseases, antiphospholipid antibody syndrome).
- Those with an expected survival of less than 2 years.
- Those known or suspected to have systemic viral, parasitic, or fungal infections, or those expected to receive antibiotic treatment within 14 days after screening.
- Those who test positive for hepatitis B surface antigen (HBsAg) or hepatitis C virus antibody (HCV Ab) or human immunodeficiency virus (HIV) antibody at screening.
- Those who have undergone liver, heart, or other solid organ transplantation or bone marrow transplantation within 1 year before screening or plan to undergo transplantation during the trial period.
- Those with a history of malignant tumors within 5 years before screening (except for cured basal cell carcinoma of the skin, squamous cell carcinoma of the skin, cervical carcinoma in situ, low-grade prostatic...
- Those with a history of drug abuse within 3 years before screening.
- Women who are pregnant or breastfeeding.
- Those with other hematopoietic system, digestive system, or central nervous system (including cerebrovascular diseases, degenerative diseases) diseases that the investigator believes will interfere with evaluation or...
- Those with severe mental illnesses that the investigator believes cannot be adequately controlled by drug treatment.
- Those unwilling to comply with the research procedures or unwilling to fully cooperate.
- Other situations deemed by the investigator as not suitable for participation in clinical trials.
The study team makes the final eligibility decision.
Where it's taking place
- Shanghai, Shanghai Municipality, China
Compensation & support
Compensation mentioned.
ClinicalTrials.gov doesn't provide a reliable structured field for payment or travel support - confirm details with the study team.
Questions & answers
Do participants get paid in this trial?
This study's listing includes signals that participants may be compensated or receive a stipend. Amounts vary and are set by the study team - confirm the details with them.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18 years to 75 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include Shanghai, Shanghai Municipality, China. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from ClinicalTrials.gov; the study team decides eligibility.