Familial Hypercholesterolemia (HoFH)
Conditions
Interventions
Experimental group:Ultrasound-guided intraperitoneal injection for the treatment of exosomes
Sponsors
Tangdu Hospital, Fourth Military Medical University
Eligibility
Sex/Gender
All
Age
18 Years to 45 Years
Inclusion criteria
Inclusion criteria: 1. Aged 18-45 years, gender is not limited; 2. Subjects with genetic diagnosis of homozygous familial hypercholesterolemia; 3. Subjects understand this research and are willing to participate in this research, and sign the informed consent.
Exclusion criteria
Exclusion criteria: 1. Have serious comorbidities, including any of the following: (1) Unstable angina and/or congestive heart failure requiring hospitalization; (2) Myocardial infarction or cerebrovascular accident within the last 6 months; (3) Chronic obstructive pulmonary disease worsens or requires hospitalization; (4) Serious vascular, nervous system, blood, gastrointestinal, endocrine system diseases or metabolic disorders; (5) Autoimmune/immunodeficiency diseases, such as rheumatoid arthritis, acquired immunodeficiency syndrome, etc.; (6) Malignant tumors or other chronic infections; 2. Received targeted drug therapy, cell therapy, gene therapy or other immunotherapy in the past; 3. Previous organ transplantation; 4. Any of the following abnormalities in laboratory examinations: (1) Blood routine: absolute neutrophil count (ANC) 1.5 x ULN; (3) Liver function: total bilirubin (TBIL) > 2 x ULN (upper limit of normal), or alanine transferase (ALT), aspartate transferase (AST), alkaline phosphatase (ALP) > 5 x ULN; (4) Renal function: serum creatinine (Cr) >= 1.5xULN, or glomerular filtration rate (GFR) < 60 mL/min*1.73m^2; (5) Echocardiography: left ventricular ejection fraction (LVEF) < 50%; 5. Those who are known or expected to have allergic reactions or have a history of allergies to any components of this trial treatment; 6. Known allergies to contrast agents; 7. There is a clear history of mental disorder in the past; 8. Past history of drug abuse or drug use; 9. Pregnant or lactating patients; 10. Women of childbearing age and men with reproductive potential cannot take effective and adequate contraceptive measures (such as IUDs, condoms, spermicidal gel plus condoms, cervical caps, etc.) during the period of receiving the study drug and 3 months after the end of the study; 11. Participated in clinical research of other drugs within 3 months before enrollment; 12. The investigator judges that the patient is not suitable to participate in this study due to other reasons.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Low density lipoprotein cholesterol; | — |
Secondary
| Measure | Time frame |
|---|---|
| Total cholesterol;Triglycerides;High-density lipoprotein cholesterol;Degree of coronary stenosis;Volume and stability of cervical vascular plaques; | — |
Countries
China
Contacts
Public ContactYuan Lijun
Outcome results
None listed