Heterozygous Familial Hypercholesterolemia (HeFH)Phase 1/2NCT07894887
EDIT-401 in Adults Who Require Additional Lowering of Low-Density Lipoprotein Cholesterol (LDL-C)
A Phase 1/2 study testing Drug: EDIT-401 in people with Heterozygous Familial Hypercholesterolemia (HeFH), run by Editas Medicine, Inc. at 2 sites in Australia.
Trial 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… Read more about this study
- Who is it for?
- Ages 18 to 65 years
- Conditions the study is for
- Heterozygous Familial Hypercholesterolemia (HeFH)
- What is being tested?
- Drug: EDIT-401
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 56 participants
- Where
- 2 sitesAustralia
- Study dates
- Nov 2026to Jul 2031
- Phase
- Phase 1/2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 65 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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
Who cannot join
- 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
Trial locations and contacts
2 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Australia2 sites
Clinical Study Center
Charlestown, New South Wales
Clinical Study Center
Perth, Western Australia
About this study
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
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).
- Being tested
- Drug: EDIT-401, Placebo
- 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
- Medical subject headings
- Hyperlipoproteinemia Type II, Coronary Artery Disease, Dyslipidemias, Lipid Metabolism Disorders, Metabolic Diseases, Nutritional and Metabolic Diseases, Lipid Metabolism, Inborn Errors, Metabolism, Inborn Errors
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Drug: EDIT-401Drug
Intravenous (IV) infusion
PlaceboOther
Intravenous (IV) infusion
| Study group | Role | What they receive |
|---|---|---|
| Cohort 1: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of EDIT-401 intravenously Receives: Drug: Drug: EDIT-401 |
| Cohort 2: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of EDIT-401 intravenously Receives: Drug: Drug: EDIT-401 |
| Cohort 3: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of EDIT-401 intravenously Receives: Drug: Drug: EDIT-401 |
| Cohort 4: Single Ascending Dose Escalation | Experimental | Participants will receive a single dose of EDIT-401 intravenously Receives: Drug: Drug: EDIT-401 |
| Placebo Comparator Study Arm | Placebo comparator | Participants will receive a single dose of EDIT-401 or Placebo intravenously Receives: Drug: Drug: EDIT-401; Other: Placebo |
| Placebo Crossover to EDIT-401 | Experimental | Participants that received placebo will receive a single dose of EDIT-401 intravenously Receives: Drug: Drug: EDIT-401 |
What is being measured
The primary outcome is the main question the study is designed to answer.
Part 1: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Measured: Up to 2 years
Part 2: Placebo-controlled percent change from baseline in serum LDL-C
Measured: Up to day 90
Secondary outcomes (7)
Part 1: Percent change from baseline in serum LDL-C
Measured: Up to day 90
Part 2: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) compared to placebo
Measured: Up to 2 year
Part 2: Proportion of participants with durable LDL-C lowering
Measured: Up to 1 year
Part 1 and Part 2: Evaluation of maximum observed concentration (Cmax)
Measured: Up to day 30
Part 1 and Part 2: Evaluation of time to maximum observed (tmax)
Measured: Up to day 30
Part 1 and Part 2: Evaluation of terminal elimination half-life (t1/2)
Measured: Up to day 30
Part 1 and Part 2: Evaluation of volume distribution (Vd)
Measured: Up to day 30
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted26 Oct 2026
- Study startNov 2026 (estimated)
- Primary completionJun 2029 (estimated)
- Study completionJul 2031 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Heterozygous Familial Hypercholesterolemia (HeFH)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Model description
- Phase 1 is an open label non-randomized single dose escalation study. Part 2 is a randomized, double-blind, placebo-controlled study with placebo crossover.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 56 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Editas Medicine, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07894887
- Sponsor's protocol ID
- EDIT-401-121-LDLR
- Status last verified
- Oct 2026
- First submitted
- 7 Oct 2026
- Last updated
- 26 Oct 2026
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT07894887
- Acronym / sponsor ID
- EDIT-401-121-LDLR
- Keywords
- EDIT-401, Gene Editing, CRISPR Editing, Familial Hypercholesterolemia, Coronary Artery Disease, Dose Escalation, LDL-C, Dyslipidemias
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Not yet recruiting
- Enrolment
- 56 participants (estimated)
- Lead sponsor
- Editas Medicine, Inc.Industry (pharma, biotech, devices)
- Study start
- Nov 2026 (estimated)
- Primary completion
- Jun 2029 (estimated)
- Study completion
- Jul 2031 (estimated)
- First posted
- 26 Oct 2026 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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).
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Sequential
- Masking
- Double blind (participant, investigator)
- Number of arms
- 6
- Model description
- Phase 1 is an open label non-randomized single dose escalation study. Part 2 is a randomized, double-blind, placebo-controlled study with placebo crossover.
| Arm | Type | Receives |
|---|---|---|
| Cohort 1: Single Ascending Dose Escalation | Experimental | Drug: Drug: EDIT-401Participants will receive a single dose of EDIT-401 intravenously |
| Cohort 2: Single Ascending Dose Escalation | Experimental | Drug: Drug: EDIT-401Participants will receive a single dose of EDIT-401 intravenously |
| Cohort 3: Single Ascending Dose Escalation | Experimental | Drug: Drug: EDIT-401Participants will receive a single dose of EDIT-401 intravenously |
| Cohort 4: Single Ascending Dose Escalation | Experimental | Drug: Drug: EDIT-401Participants will receive a single dose of EDIT-401 intravenously |
| Placebo Comparator Study Arm | Placebo comparator | Drug: Drug: EDIT-401, Other: PlaceboParticipants will receive a single dose of EDIT-401 or Placebo intravenously |
| Placebo Crossover to EDIT-401 | Experimental | Drug: Drug: EDIT-401Participants that received placebo will receive a single dose of EDIT-401 intravenously |
Who may qualify
- Ages
- 18 to 65 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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
Key exclusion criteria (7 listed)
- 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):
- Active or chronic history of liver disease
- Reduced renal function
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Drug: EDIT-401Intravenous (IV) infusion | Drug | Cohort 1: Single Ascending Dose Escalation, Cohort 2: Single Ascending Dose Escalation, Cohort 3: Single Ascending Dose Escalation, Cohort 4: Single Ascending Dose Escalation, Placebo Comparator Study Arm, Placebo Crossover to EDIT-401 |
| PlaceboIntravenous (IV) infusion | Other | Placebo Comparator Study Arm |
Primary outcome
Part 1: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs)
Time frame: Up to 2 years
Part 2: Placebo-controlled percent change from baseline in serum LDL-C
Time frame: Up to day 90
7 secondary outcomes
Part 1: Percent change from baseline in serum LDL-C
Time frame: Up to day 90
Part 2: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) compared to placebo
Time frame: Up to 2 year
Part 2: Proportion of participants with durable LDL-C lowering
Time frame: Up to 1 year
Part 1 and Part 2: Evaluation of maximum observed concentration (Cmax)
Time frame: Up to day 30
Part 1 and Part 2: Evaluation of time to maximum observed (tmax)
Time frame: Up to day 30
Part 1 and Part 2: Evaluation of terminal elimination half-life (t1/2)
Time frame: Up to day 30
Part 1 and Part 2: Evaluation of volume distribution (Vd)
Time frame: Up to day 30
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 26 Oct 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Drug: EDIT-401 in people with Heterozygous Familial Hypercholesterolemia (HeFH), run by Editas Medicine, Inc. at 2 sites in Australia.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: opening soon, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in Australia. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Daniel Ory, MD (617-401-9000, patients@editasmed.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Part 1: Incidence of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) (time frame: Up to 2 years). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 26 Oct 2026.