Exosome-based Nanoplatform for Ldlr mRNA Delivery in FH

Trial statusNot yet recruiting
Trial phasePhase 1
Trial typeInterventional
Biological sexAll
Age18-45
SponsorTang-Du Hospital

About this trial

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.

Eligibility criteria

Qualifiers

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

Disqualifiers

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 previously received targeted drug therapy, cell therapy, gene therapy or others immunotherapy;

those who had organ transplants in the past;

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%.

Trial design

Treatments tested in this trial

  • Low Density Lipoprotein Receptor mRNA Exosomes

Treatment groups

30 Participants
are divided into 1 treatment group

Sponsors and collaborators

Tang-Du Hospital

Lead sponsor

Air Force Military Medical University, China

Collaborator