EDIT-401 gene editing for adults with heterozygous familial hypercholesterolemia and high LDL cholesterol
Tests EDIT-401 gene editing to lower LDL cholesterol in adults with familial hypercholesterolemia.
Plain-language title generated from the registry record
Registry title: EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
Key facts
- Where
- Charlestown and Perth, AustraliaSee the 2 sites
- Phase
- Phase 1/2
Could this be for you?
Answer for yourself. This is not a decision: only the study team can say whether you can take part.
- Are you between 18 and 65 years old?
Source: NCT07894887, who can take part
Where in Australia
- Clinical Study CenterCharlestown, AustraliaNo longer taking part
- Clinical Study CenterPerth, AustraliaNo longer taking part
Questions to ask the study team
- How often do I need to come in, and for how long each time?
- Are travel costs paid?
- Can I keep my own doctor?
- What happens if I want to stop?
Trial details
- Who runs it
- Editas Medicine, Inc.
- Trial code
- NCT07894887
- Record updated
- 26 October 2026
- Original description
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).Read full original descriptionShow less
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
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).
Inclusion: 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.
Exclusion: 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; Reduced renal function.