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NOT YET RECRUITING
NCT07894887
PHASE1/PHASE2

EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)

Sponsor: Editas Medicine, Inc.

View on ClinicalTrials.gov

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

Drug: EDIT-401

Intravenous (IV) infusion

OTHER

Placebo

Intravenous (IV) infusion

Locations (2)

Clinical Study Center

Charlestown, New South Wales, Australia

Clinical Study Center

Perth, Western Australia, Australia