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Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH) | Clinical Trials | Clareo Health

RECRUITINGEARLY_PHASE1

Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)

NCT06461702•The First Affiliated Hospital of Bengbu Medical University

View on ClinicalTrials.gov

Summary

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. OBD ≤ Maximum Tolerable Dose (MTD).

Detailed Description

In this study, the longest screening period for the main study was 42 days, the treatment day was Day 1 (D1), and the safe follow-up period was up to 52 weeks after medication. In the main study, when OBD occurs, additional subjects will be added to the dose group (specific number of cases will be negotiated between the cooperating organization and investigators) for further validation. In addition, subjects in the first dose group can voluntarily receive a second drug administration of OBD level. After the completion of the main study, participants will undergo long-term follow-up. According to the Technical Guidelines for Long term Follow up Clinical Research of Gene Therapy Products (Trial) released by CDE, a long-term follow-up until 15 years after the medicine administration is required .

Eligibility

Age

18 - 65 years

Sex

ALL

Healthy Volunteers

Inclusion Criteria

•1. Men or women aged ≥ 18 years and ≤ 65 years (including boundary values) who signed informed consent.
•2. Meets the diagnostic criteria for familial hypercholesterolemia. When screening, there are mutations in the PCSK9 and/or ApoB and/or LDLR genes.
•4\. When screening, the weight should be ≥ 40kg, and the body mass index (BMI) should be between 18-30 kg/m2 (including boundary values).
•5\. During screening, subjects must meet the following laboratory standards: 5.1 Blood routine: Absolute neutrophil count (ANC) ≥ 1.5 × 109/L, platelet count (PLT) ≥ 100 × 109/L, hemoglobin count (HGB) ≥ 90 g/dL; 5.2 Liver function: aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP) \< 2.0 × ULN, total bilirubin (TBIL) ≤ 1.5 × ULN; 5.3 Renal function: Serum creatinine (Cr) ≤ 1.5 × ULN, and glomerular filtration rate (GFR)\>60mL/min \* 1.73m2; 5.4 Coagulation function: prothrombin time (PT), activated partial thromboplastin time (APTT), international standardized ratio (INR)\<1.5 x ULN; 5.5 Low density lipoprotein cholesterol (LDL-C) ≥ 4.21mmol/L, and fasting triglycerides\<5.6mmol/L.
•6\. The subjects and their partners must take effective contraceptive measures during the participation in this study until 6 months after the end of the main study.
•7\. The subject must agree not to accept other lipid-lowering drugs for at least 28 days after receiving the investigational drug.
•8\. Voluntarily sign informed consent.

Exclusion Criteria

•1. Those who have used any prescription drugs that affect blood lipid metabolism within at least 14 days before receiving the study drug (or within 7 half lives of the drug, whichever is longer, applicable to small molecule/small nuclear acid drugs) or within 3 months (applicable to biological agents such as PCSK9 inhibitors), or who have used any over-the-counter drugs that affect blood lipid metabolism within at least 14 days before receiving the study drug (such as Chinese medicine/traditional Chinese patent medicines and simple preparations, vitamins, fish oil (\>1000mg/day), drugs containing red koji rice or health products, etc.), (Exception of those who have received stable non-cyclical hormone replacement therapy for more than 8 weeks and agree not to change the hormone treatment more than 28 days after the study drug administration); individuals who have participated in clinical studies of other lipid-lowering drugs and have accepted the investigational drugs within 6 months before the screening period.
•2. Poorly controlled hypertensive patients who have receive conventional treatments (systolic blood pressure (SBP) ≥ 160mmHg and/or diastolic blood pressure (DBP) ≥ 100mmHg).
•3. Poorly controlled diabetes patients (glycosylated hemoglobin\>8.5%).
•4. Individuals who are allergic to drugs or mRNA vaccine components contained in lipid nanoparticles (LNPs), or have experienced adverse reactions to LNP drug therapy, such as:
•4.1 After receiving LNP drug treatment, ALT or AST\>3.0 × ULN; 4.2 After receiving LNP drug treatment, INR\>1.5 or APTT/d-dimer\>1.5 × ULN; 4.3 Any infusion response that requires clinical intervention, slows down infusion rate, or stops LNP drugs treatment; 4.4 Any other adverse reactions that researchers believe are related to the treatment of LNP drugs.
•5. Within three months before the screening, individuals who smoke more than 5 cigarettes per day or consume an equal amount of nicotine or nicotine substitutes.
•6. Individuals with a history of alcohol abuse \[consuming more than 14 units of alcohol per week (1 unit ≈ 360mL of beer or 45mL of 40% liquor or 150mL of wine)\] within 3 months before the screening; Individuals positive for alcohol breath test during the screening or admission.
•7. Grade III-IV heart failure defined by the New York Heart Association (NYHA), or left ventricular ejection fraction\<50%, or prolonged QTc interval (\>470ms in females and\>450ms in males) found during the screening.
•8. Suffering poorly controlled severe arrhythmia , such as recurrent and highly symptomatic ventricular tachycardia with poorly drug controlled, atrial fibrillation with rapid ventricular reaction, or supraventricular tachycardia within three months before the screening.
•9. Myocardial infarction, unstable angina, percutaneous coronary intervention, coronary artery bypass grafting, severe deep vein thrombosis or pulmonary embolism within three months before the screening; Cerebrovascular accidents occurring within 6 months before the screening or planning for cardiac surgery or revascularization during the main study period.
+16 more criteria

Locations (1)

The First Affiliated Hospital of Bengbu Medical College

Bengbu, Anhui, China

Key Dates

Start Date

April 1, 2024

Primary Completion Date

August 1, 2025

Completion Date

December 1, 2025

Last Updated

June 17, 2024

Enrollment

ESTIMATED participants

Conditions

Familial Hypercholesterolemia

Interventions

YOLT-101

DRUG

Contacts

Wang Hongju

CONTACT

13955231336 1649134019@qq.com

Zhou Huan

CONTACT

13665527160 zhouhuanbest@163.com

A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)

NCT07058077

Heterozygous Familial Hypercholesterolemia (HeFH)

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The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

NCT01109368

Homozygous Familial Hypercholesterolemia

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A Study on Efficacy and Safety of HST101 in Chinese Patients with Hypercholesterolemia

Data Source & Attribution

This clinical trial information is sourced from ClinicalTrials.gov, a service of the U.S. National Institutes of Health.

ClinicalTrials.gov last update: June 17, 2024Data synced to Clareo: March 29, 2026

Modifications: This data has been reformatted for display purposes. Eligibility criteria have been parsed into inclusion/exclusion sections. Location data has been geocoded to enable distance-based search. For the authoritative and most current information, please visit ClinicalTrials.gov.

Neither the United States Government nor Clareo Health make any warranties regarding the data. Check ClinicalTrials.gov frequently for updates.

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Clinical Exploration Trial of YOLT-101 in the Treatment of Familial Hypercholesterolemia (FH)

Inclusion Criteria

Exclusion Criteria

A Study of Enlicitide Decanoate (MK-0616, an Oral PCSK9 Inhibitor) in Children and Adolescents With Heterozygous Familial Hypercholesterolemia (MK-0616-029)

The Rogosin Institute Homozygous Familial Hypercholesterolemia Repository

A Study on Efficacy and Safety of HST101 in Chinese Patients with Hypercholesterolemia

Familial Hypercholesterolemia Canada / Hypercholesterolemie Familiale Canada

Collaborative Approach to Reach Everyone With Familial Hypercholesterolemia (CARE-FH)

Evaluation of the Effect of Lomitapide Treatment on Major Adverse Cardiovascular Events (MACE) in Patients With Homozygous Familial Hypercholesterolemia