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EDIT-401

Phase 1

Heterozygous Familial Hypercholesterolemia (HeFH) | Small molecule | Metabolic |Editas Medicine, Inc.|Trials Updated: Oct 26, 2026

Success Probability

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Market & Valuation

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Trial Design

Double-BlindPLACEBO_CONTROLLED
Total Trials1
Total Enrollment56

FDA Designations

No designations recorded

EDIT-401 clinical trials

EDIT-401 · 1 trial · 1 indication

Phase 1 1
NCT07894887EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)Heterozygous Familial Hypercholesterolemia (HeFH)
NOT YET_RECRUITING56 Analytics
PHASE1NOT YET_RECRUITING
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Heterozygous Familial Hypercholesterolemia (HeFH)
NCT0789488756
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Study Endpoints

Primary Endpoints

Part 1: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Up to 2 years
Part 2: Placebo-controlled percent change from baseline in serum LDL-C
Up to day 90

Secondary Endpoints

Part 1: Percent change from baseline in serum LDL-C
Up to day 90
Part 2: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) compared to placebo
Up to 2 year
Part 2: Proportion of participants with durable LDL-C lowering
Up to 1 year
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Study Design & Arms

AllocationNON_RANDOMIZED
MaskingDOUBLE
ModelSEQUENTIAL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
Cohort 1: Single Ascending Dose EscalationEXPERIMENTALParticipants will receive a single dose of EDIT-401 intravenously
Cohort 2: Single Ascending Dose EscalationEXPERIMENTALParticipants will receive a single dose of EDIT-401 intravenously
Cohort 3: Single Ascending Dose EscalationEXPERIMENTALParticipants will receive a single dose of EDIT-401 intravenously
Cohort 4: Single Ascending Dose EscalationEXPERIMENTALParticipants will receive a single dose of EDIT-401 intravenously
Placebo Comparator Study ArmPLACEBO_COMPARATORParticipants will receive a single dose of EDIT-401 or Placebo intravenously
Placebo Crossover to EDIT-401EXPERIMENTALParticipants that received placebo will receive a single dose of EDIT-401 intravenously

Interventions

NameTypeDescription
Drug: EDIT-401DRUGIntravenous (IV) infusion
PlaceboOTHERIntravenous (IV) infusion
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Eligibility Criteria

Age Range18 Years to 65 Years
SexALL
Healthy VolunteersNo
Study Sites2

Key inclusion Criteria: 1. Clinical diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH) 2. Low-density lipoprotein cholesterol (LDL-C) ≥ 70 and ≤ 300 mg/dL (≥ 1.8 mmol/L and ≤ 7.8 mmol/L) 3. Treatment including two (2) or more approved Lipid Lowering Therapies 4. Participant weight \> 40...

Countries:Australia
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Recent Changes (Last 90 Days)

LOWOct 26, 2026NCT07894887NEW_TRIAL: changed
LOWOct 26, 2026NCT07894887NEW_TRIAL: changed

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