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LCAT gene-transduced autologous preadipocyte transplantation in familial LCAT deficiency (24-week safety study)
jRCT2033200096Phase 1Recruiting
- Sponsor
- Japan Agency for Medical Research and Development
- Start
- Aug 2020
- Modality
- Cell Therapies, Gene Therapies
- Countries
- 1
01
Overview
Phase 1, open-label, single-arm trial of LCAT gene-transduced autologous human preadipocytes in Familial lecithin:cholesterol acyltransferase deficiency (FLD), sponsored by Japan Agency for Medical Research and Development. Primary endpoints: Occurrence of adverse events related to the clinical trial product, Appearance of replication-competent retrovirus (RCR) and 3 more. Started Aug 2020; status: recruiting.
02
Drugs
LCAT gene-transduced autologous human preadipocytes
03
Conditions
Familial lecithin:cholesterol acyltransferase deficiency (FLD)
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