Tests exosome-carried LDLR mRNA therapy for familial hypercholesterolemia

Trial details

Who runs it

Tang-Du Hospital

Trial code

NCT05043181

Record updated

14 September 2021

Original description

mRNA therapy is a highly promising gene therapeutic strategy in the treatment of Homozygous Familial Hypercholesterolemia (HoFH). Exosomes is safe and efficient carriers for mRNA drug delivery, due to their biocompatibility, bioavailability. This first-in-human study is aimed to evaluate the safety and preliminary effectiveness of Exosome-based ldlr mRNA nanoplatform for gene therapy in HoFH.Read full original descriptionShow less

Exosome-based Nanoplatform for Ldlr mRNA Delivery in Familial Hypercholesterolemia

mRNA therapy is a highly promising gene therapeutic strategy in the treatment of Homozygous Familial Hypercholesterolemia (HoFH). Exosomes is safe and efficient carriers for mRNA drug delivery, due to their biocompatibility, bioavailability. This first-in-human study is aimed to evaluate the safety and preliminary effectiveness of Exosome-based ldlr mRNA nanoplatform for gene therapy in HoFH.

Inclusion: Age 18-45, no gender limitation; Patients with homozygous familial hypercholesterolemia diagnosed by genetic testing; Understand the study and be willing to participate in the study with the informed consent signed.

Exclusion: those who previously received targeted drug therapy, cell therapy, gene therapy or others immunotherapy; Women of childbearing age and fertile men cannot take effective and adequate contraceptive measures (such as intrauterine device (IUD), condom, spermicidal gel plus condom, uterine cap, etc.) during the period of receiving the study drug and 3 months after the end of the study; those who participated in the clinical study of other drugs within 3 months before joining the group; those who have severe comorbidities, including any of the following: A) unstable angina pectoris and/or congestive heart failure requiring hospitalization; B) myocardial infarction or cerebrovascular accident within the last 6 months; C) chronic obstructive pulmonary disease worsens or requires hospitalization; D) serious diseases of vascular, nervous system, blood, gastrointestinal and endocrine systems or metabolic disorders; E) autoimmune/immune deficiency diseases such as rheumatoid arthritis, acquired immune deficiency syndrome, and so on; F) malignant tumor or other chronic infection; those who are known or expected to have an allergic reaction to or have a history of allergic reaction to any of the ingredients treated by this test; those who have a history of contrast agent allergic; those who have a clear history of mental disorders in the past; those who have a history of drug abuse or drug use; any of the following abnormalities are found in the laboratory examination: A) blood routine examination: absolute neutrophil count (ANC) < 1.5×109/L, or platelet (PLT) < 50×109/L, or hemoglobin (HGB) < 80 g/dL; B) coagulation function: prothrombin time (PT), or activated partial thrombin time (APTT), or INR > 1.5×ULN; C) liver function: total bilirubin (TBIL) > 2×ULN (upper limit of normal value), or alanine transferase (ALT), aspartate transferase (AST), alkaline phosphatase (ALP) > 5×ULN; D) renal function: serum creatinine (Cr) ≥1.5×ULN, or glomerular filtration rate (GFR) < 60 mL/min·1.73m2; E) cardiac ultrasound: left ventricular ejection fraction (LVEF) < 50%; those who had organ transplants in the past; Pregnant or lactating women; Subjects that are not suitable to participate in this study for other reasons judged by the investigators.

ClinicalTrials.gov NCT05043181