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EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Sponsor: Editas Medicine, Inc.
Summary
A phase 1/2, two-part trial consisting of an open label dose-escalation part and a randomized, double blind, placebo-controlled part to investigate safety, tolerability, pharmacokinetics, and efficacy with EDIT-401 gene editing in adult male and female participants 18 to 65 years of age who require additional lowering of low-density lipoprotein cholesterol (LDL-C) such as those with clinical diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH).
Official title: Phase 1/2 Trial to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy of EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol
Key Details
Gender
All
Age Range
18 Years - 65 Years
Study Type
INTERVENTIONAL
Enrollment
56
Start Date
2026-11
Completion Date
2031-07
Last Updated
2026-10-26
Healthy Volunteers
No
Interventions
Drug: EDIT-401
Intravenous (IV) infusion
Placebo
Intravenous (IV) infusion
Locations (2)
Clinical Study Center
Charlestown, New South Wales, Australia
Clinical Study Center
Perth, Western Australia, Australia