drugset / Trial / NCT05043181
Exosome-based Nanoplatform for Ldlr mRNA Delivery in FH
Phase 1
Not yet recruiting
30 enrolled
Tang-Du Hospital
Air Force Military Medical University, China · collab
NaSingle-groupOpen-labelTreatment
Summary
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.
Timeline
- Start
- 2021-12
- Primary completion
- 2026-12
- Completion
- 2026-12
Drugs
| Evaluation | Drug | Modality | Dose | Route |
|---|---|---|---|---|
| Subject | LDLR mRNA exosomes | Unknown | 0.044 mg/kg | Intravenous |
| Subject | LDLR mRNA exosomes | Unknown | 0.088 mg/kg | Intravenous |
| Subject | LDLR mRNA exosomes | Unknown | 0.145 mg/kg | Intravenous |
| Subject | LDLR mRNA exosomes | Unknown | 0.22 mg/kg | Intravenous |
| Subject | LDLR mRNA exosomes | Unknown | 0.295 mg/kg | Intravenous |
| Subject | LDLR mRNA exosomes | Unknown | 0.394 mg/kg | Intravenous |