NCT07894887Not yet recruitingPhase 1/2
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
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
Source: ClinicalTrials.gov · Last updated 26 Oct 2026
Brief summary (registry, in English)
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).
- Status (registry)
- Not yet recruiting
- Phase (registry)
- Phase 1/2
- Study type
- Interventional
- Enrollment
- 56 (estimated)
- First submitted
- 7 Oct 2026
- Start date
- Nov 2026
- Primary completion date
- Jun 2029
- Lead sponsor
- Editas Medicine, Inc.
- NCT number
- NCT07894887
What you commit to
- Study type: Interventional · Phase 1/2
- Planned participants: 56
- From Nov 2026 to the primary completion date, Jun 2029
- 2 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Other illness
- Any known history of Homozygous Familial Hypercholesterolemia (HoFH) or lipid apheresis
- Evidence of an unstable or acute cardiovascular disorder
- Any history of Diabetes Mellitus (DM):
- Part 1: Type 1 and Type 2 DM
- Part 2: Type 1 DM
- Active or chronic history of liver disease
Other
- Reduced renal function
Who the study is looking for
- Clinical diagnosis of Heterozygous Familial Hypercholesterolemia (HeFH)
- Low-density lipoprotein cholesterol (LDL-C) ≥ 70 and ≤ 300 mg/dL (≥ 1.8 mmol/L and ≤ 7.8 mmol/L)
- Treatment including two (2) or more approved Lipid Lowering Therapies
- Participant weight \> 40 kg and ≤ 110 kg
Age in the registry: 18 years to 65 years · Sex: All · Does not accept healthy volunteers
Sites (2)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| Clinical Study Center Coordinator of this site? Confirm its status | Charlestown | Australia | Unknown status |
| Clinical Study Center Coordinator of this site? Confirm its status | Perth | Australia | Unknown status |
This page is information, not medical advice. Discuss any trial with your doctor.