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Not yet recruitingNCT07894887Updated Oct 26, 2026

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

A Phase 1/2 interventional study of Drug: EDIT-401 and Placebo in Heterozygous Familial Hypercholesterolemia (HeFH), sponsored by Editas Medicine, Inc.. Not yet recruiting at 2 sites in Australia. Open to participants aged 18 Years to 65 Years. Per ClinicalTrials.gov, last updated 2026-10-26.

Sponsored by Editas Medicine, Inc. · Phase 1/2, Interventional, and Treatment

Updated Oct 26, 2026Newly registeredGo to Updates ↓
Phase
Phase 1/2
Study type
Interventional
Enrollment
56
Allocation
Non-randomized
Ages
18 Years to 65 Years
Sex
All
01

Study 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).

02

Conditions studied

  • Heterozygous Familial Hypercholesterolemia (HeFH)

Keywords

  • EDIT-401
  • Gene Editing
  • CRISPR Editing
  • Familial Hypercholesterolemia
  • Coronary Artery Disease
  • Dose Escalation
  • LDL-C
  • Dyslipidemias
  • Lipid Metabolism Disorders
  • Metabolic Diseases
  • Nutritional and Metabolic Diseases
  • Lipid Metabolism, Inborn Errors
  • Metabolism, Inborn Errors
  • Genetic Diseases, Inborn
  • Congenital, Hereditary, and Neonatal Diseases and Abnormalities
  • Hyperlipoproteinemias
  • Hypercholesterolemia
  • Hyperlipidemias
  • Hyperlipoproteinemia Type II
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In context

Hyperlipoproteinemia Type II

249 studies on the registry are indexed under Hyperlipoproteinemia Type II; 51 are open to participants now.

This study's planned enrollment of 56 is below the median of 93 across 173 interventional studies indexed under Hyperlipoproteinemia Type II.

Browse Hyperlipoproteinemia Type II studies →

Lead sponsor

Editas Medicine, Inc. is the lead sponsor of 6 studies on the registry; 2 are open to participants now.

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
18 Years to 65 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

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 kg and ≤ 110 kg

Key Exclusion Criteria:

  1. Any known history of Homozygous Familial Hypercholesterolemia (HoFH) or lipid apheresis
  2. Evidence of an unstable or acute cardiovascular disorder

  3. Any history of Diabetes Mellitus (DM):

    1. Part 1: Type 1 and Type 2 DM
    2. Part 2: Type 1 DM
  4. Active or chronic history of liver disease
  5. Reduced renal function
05

Study design

Phase
Phase 1 / Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Sequential assignment
Masking
Double (Participant, Investigator)
Enrollment
56 participants (estimated)

Study arms

  • Experimental
    Cohort 1: Single Ascending Dose Escalation

    Participants will receive a single dose of EDIT-401 intravenously

    Drug: Drug: EDIT-401

  • Experimental
    Cohort 2: Single Ascending Dose Escalation

    Participants will receive a single dose of EDIT-401 intravenously

    Drug: Drug: EDIT-401

  • Experimental
    Cohort 3: Single Ascending Dose Escalation

    Participants will receive a single dose of EDIT-401 intravenously

    Drug: Drug: EDIT-401

  • Experimental
    Cohort 4: Single Ascending Dose Escalation

    Participants will receive a single dose of EDIT-401 intravenously

    Drug: Drug: EDIT-401

  • Placebo comparator
    Placebo Comparator Study Arm

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

    Drug: Drug: EDIT-401 · Other: Placebo

  • Experimental
    Placebo Crossover to EDIT-401

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

    Drug: Drug: EDIT-401

Interventions

  • DrugDrug: EDIT-401

    Intravenous (IV) infusion

  • OtherPlacebo

    Intravenous (IV) infusion

06

What researchers measure

Primary outcomes

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

    Time frame: Up to 2 years

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

    Time frame: Up to day 90

Secondary outcomes

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

    Time frame: Up to day 90

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

    Time frame: Up to 2 year

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

    Time frame: Up to 1 year

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

    Time frame: Up to day 30

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

    Time frame: Up to day 30

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

    Time frame: Up to day 30

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

    Time frame: Up to day 30

07

Study locations

2 sites
  • Clinical Study Center
    Charlestown, New South Wales, Australia
  • Clinical Study Center
    Perth, Western Australia, Australia
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

1 registry update since Sep 25, 2026
Registered
First appeared on the registry. No changes since
Oct 26, 2026
Show all 1 update
  1. Oct 26, 2026
    First appeared on the registry

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT07894887
Lead sponsor
Editas Medicine, Inc.
Responsible party
Sponsor
First posted
Oct 26, 2026
Start date
Nov 2026 (estimated)
Primary completion
Jun 2029 (estimated)
Completion
Jul 2031 (estimated)
Last update
Oct 26, 2026

Study contacts

Daniel Ory, MD
Contact
patients@editasmed.com
617-401-9000

Oversight

FDA-regulated drug
No
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

Not currently enrolling

This study is not yet recruiting, as verified in Oct 2026. You cannot join it, but the record below documents what was studied.

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