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Trial intelligence / Profile preview
AAV8-hLDLR gene replacement for homozygous familial hypercholesterolemia
NL-OMON47467Active, not recruiting
- Sponsor
- REGENXBIO Inc.
- Start
- Dec 2017
- Modality
- Gene Therapies
- Countries
- 1
01
Overview
Open-label, single-arm trial of AAV-hLDLR in homozygous familial hypercholesterolemia (HoFH), sponsored by REGENXBIO Inc. Primary endpoint: Safety up to 24 weeks. Started Dec 2017; status: active, not recruiting.
02
Drugs
AAV-hLDLR
03
Conditions
homozygous familial hypercholesterolemia (HoFH)
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