NCT07894887

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

StatusNot Yet Recruiting

PhasePhase 1/2

SponsorEditas Medicine, Inc.

Primary completionJun 2029 (estimated)

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Record History

All 1 version: 1 with changes below.

Oct 21, 2026 Study First Submitted

Endpoints

Primary

Part 1: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)

Timeframe: Up to 2 years

Part 2: Placebo-controlled percent change from baseline in serum LDL-C

Timeframe: Up to day 90

Secondary (7)

Part 1: Percent change from baseline in serum LDL-C

Part 2: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) compared to placebo

Part 2: Proportion of participants with durable LDL-C lowering

Part 1 and Part 2: Evaluation of maximum observed concentration (Cmax)

Part 1 and Part 2: Evaluation of time to maximum observed (tmax)

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Part 1 and Part 2: Evaluation of terminal elimination half-life (t1/2)

Part 1 and Part 2: Evaluation of volume distribution (Vd)

Arms and interventions

Cohort 1: Single Ascending Dose EscalationExperimental

Participants will receive a single dose of EDIT-401 intravenously

Cohort 2: Single Ascending Dose EscalationExperimental

Participants will receive a single dose of EDIT-401 intravenously

Cohort 3: Single Ascending Dose EscalationExperimental

Participants will receive a single dose of EDIT-401 intravenously

Cohort 4: Single Ascending Dose EscalationExperimental

Participants will receive a single dose of EDIT-401 intravenously

Placebo Comparator Study ArmPlacebo comparator

Participants will receive a single dose of EDIT-401 or Placebo intravenously

Placebo Crossover to EDIT-401Experimental

Participants that received placebo will receive a single dose of EDIT-401 intravenously

Drugs: Drug: EDIT-401

Other: Placebo

Sites

2 sites in 1 country

About this trial

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).

Trial details

Enrollment: 56 participants (estimated)
Started: Nov 2026
Study completion: Jul 2031 (estimated)
Eligibility: 18 to 65, all sexes, no healthy volunteers
Design: Interventional, non-randomized, double blind, treatment
Conditions: Congenital, Hereditary, and Neonatal Diseases and Abnormalities, Coronary Artery Disease, Dyslipidemias, Genetic Diseases, Inborn, Hypercholesterolemia, Hyperlipidemias, Hyperlipoproteinemia Type II, Hyperlipoproteinemias +6 more
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
First posted: Oct 26, 2026
Registry updated: Oct 26, 2026
Last verified: Oct 2026

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