CardiovascularPhase 1NCT07491172
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
A Phase 1 study testing CTX310 in people with Cardiovascular and Metabolic Disease, run by CRISPR Therapeutics AG at 17 sites in 4 countries.
Trial summary
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Cardiovascular, Metabolic Disease, Dyslipidemias
- What is being tested?
- CTX310
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 90 participants
- Where
- 17 sitesin 4 countries
- Study dates
- 21 Jun 2024to Jun 2028
- Phase
- Phase 1
- 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 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Age of ≥18 and ≤75 years at the time of signing the informed consent.
- Able to provide written informed consent.
- Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL.
- Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening
- Female participants must be postmenopausal or surgically sterile.
- All male participants and their female partners must agree to the use of an acceptable method of effective contraception for the duration of the study.
Who cannot join
- Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
- Evidence of liver disease, defined as but not limited to:
- LFTS >2 × upper limit of normal (ULN), or total bilirubin >2 × ULN, or INR >1.5 × ULN, or liver stiffness measured by liver elastography
- Abnormal or compromised function of kidney, heart, blood or liver.
- Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.
- Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran).
Show all 11Show fewer
- Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial.
- Any prior malignancy within the past 5 years, or current malignancy (exceptions for resected or removed basal cell carcinoma, squamous cell carcinoma in situ and carcinoma in situ of the cervix or breast).
- Women of childbearing potential.
- Note: Other protocol defined Inclusion/Exclusion criteria may apply.
- Note: The inclusion and exclusion criteria listed represent the global protocol. Additional or modified eligibility criteria may apply in certain countries in accordance with local regulatory and ethics committee requirements and the approved country-specific protocol.
Trial locations and contacts
17 sites in 4 countries. Contact the study team or a site near you to ask about taking part.
17 sites in 4 countries
United States8 sites, 8 recruiting
- Recruiting
Duke Early Phase Clinical Research
Durham, North Carolina, 27710
Debbie Freeman, 919-613-1223, debra.h.freeman@duke.edu
- Recruiting
Integrity Clinical Research
Hialeah, Florida, 33015
Celia Padron, 786-953-6263, cpadronmd@integrityresearchcenter.com
- Recruiting
Peters Medical Research
High Point, North Carolina, 27260
Erika Jordan, 336-883-9773, erikaj@petersmedicalresearch.com
- Recruiting
Jacksonville Center for Clinical Research
Jacksonville, Florida, 32216
Sonia Gerardo, 904-730-0101
- Recruiting
Flourish Orlando
Orlando, Florida, 32789
Stephanie Munoz, 407-740-8078, smunoz@flourishresearch.com
- Recruiting
United Medical Research
Port Orange, Florida, 32127
Bhanu Visalingam, 386-523-9498, drvisvalingam@progressivemedicalresearch.com
- Recruiting
Oregon Health & Science University (OHSU)
Portland, Oregon, 97239
Hailey Volk, 503-494-2004, volkh@ohsu.edu
- Recruiting
University of Rochester
Rochester, New York, 14642
Jeffrey Bruckel, 585-275-2475, Jeffrey_Bruckel@URMC.Rochester.edu
Australia5 sites, 5 recruiting
- Recruiting
Royal Adelaide
Adelaide, 5000
Denise Healy, +61 409 877 807, Denise.Healy@sa.gov.au
- Recruiting
CORE Research
Brisbane, 4064
Daunte Tamaki, 07 + 38765688, daunte.tamaki@coreresearchgroup.com
- Not yet recruiting
Royal Prince Alfred Hospital
Camperdown, 2050
Aimei Lee, (02) + 9515 4860, aimei.lee@health.nsw.gov.au
- Recruiting
Austin Health
Heidelberg, 3084
Adele Manzoney, +613 9496 2160, adele.manzoney@unimelb.edu.au
- Recruiting
Monash Medical Center
Melbourne, 3168
Debra O'Leary, +61 3 75111243, debra.oleary@monashhealth.org
New Zealand2 sites, 2 recruiting
- Recruiting
Aotearoa
Auckland, 2025
Kate Msiska, +64 (0) 21 850 235, kate.msiska@aotearoatrials.nz
- Recruiting
NZCR
Christchurch, 8011
Jo Sanders, +64 (0) 3 372 9477, jo.sanders@nzcr.co.nz
United Kingdom2 sites, 2 recruiting
- Recruiting
Royal Papworth Hospital
Cambridge
Jenny Castedo, +441223 638000, jenny.castedo1@nhs.net
- Recruiting
Richmond Pharmacology
London, SE1 1YR
Volunteer Recruitment Team, +44 (0)20 7042 5800, volunteer@richmondpharmacology.com
About this study
Official title: A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.
Read the detailed description
This is a phase 1, open-label, multi-center study of CTX310 in participants with refractory dyslipidemias. Subjects will receive a dose of CTX310 via intravenous (IV) infusion.
- Conditions
- Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH)
- Being tested
- CTX310
- Keywords
- Refractory Dyslipidemias
- Medical subject headings
- Metabolic Diseases, Dyslipidemias, Lipid Metabolism Disorders, Hypertriglyceridemia, Homozygous Familial Hypercholesterolemia, Hyperlipoproteinemia Type V, Hypercholesterolemia
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
CTX310Drug
CTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3).
| Study group | Role | What they receive |
|---|---|---|
| CTX310 | Experimental | Subjects will receive an intravenous (IV) infusion. Receives: Drug: CTX310 |
What is being measured
The primary outcome is the main question the study is designed to answer.
To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments
Incidence of dose-limiting toxicities and frequency of adverse events
Measured: From CTX310 infusion up to 12 months
Secondary outcomes (4)
To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Percentage change from baseline in lipid values (LDL-C, non-HDL-C, Trigs, apoB and HDL-C)
Measured: Over 12 months, compared to baseline
To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Frequency and severity of adverse events (AE), including treatment-emergent adverse events (TEAEs) and adverse events of special interest (AESIs), clinically significant laboratory abnormalities, and clinically significant abnormal vital signs.
Measured: From CTX310 infusion up to 12 months
To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Levels of CTX310 in blood over time
Measured: From CTX310 infusion up to 12 months
To assess the pharmacodynamic (PD) response of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Percentage change from baseline of ANGPTL3
Measured: Over 12 months, compared to baseline
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Mar 2026
- Study start21 Jun 2024
- Primary completionJun 2027 (estimated)
- Study completionJun 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for CardiovascularStudies 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
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- CRISPR Therapeutics AG
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07491172
- Sponsor's protocol ID
- CRSP-CVD-400
- Status last verified
- Sep 2026
- First submitted
- 18 Mar 2026
- Last updated
- 18 Sep 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Sep 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 1 Open-label, Multicenter, First-in-human, Ascending Dose Trial Evaluating the Safety and Tolerability of a Lipid Nanoparticle Formulation of CRISPR-Guide RNA-Cas9 Nuclease (CTX310) for In Vivo Editing of the Angiopoietin-like 3 (ANGPTL3) Gene in Participants With Refractory Dyslipidemias
- ClinicalTrials.gov ID
- NCT07491172
- Acronym / sponsor ID
- CRSP-CVD-400
- Conditions
- Cardiovascular, Metabolic Disease, Dyslipidemias, Lipid Disorder, Hypertriglyceridemia, Heterozygous Familial Hypercholesterolemia (HeFH)
- Keywords
- Refractory Dyslipidemias
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- CRISPR Therapeutics AGIndustry (pharma, biotech, devices)
- Study start
- 21 Jun 2024 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jun 2028 (estimated)
- First posted
- 24 Mar 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.
This is a single-arm, open-label, multicenter, ascending dose Phase 1 trial that will enroll participants 18 to 75 years of age with dyslipidemias that are refractory to available treatments.
Detailed description
This is a phase 1, open-label, multi-center study of CTX310 in participants with refractory dyslipidemias. Subjects will receive a dose of CTX310 via intravenous (IV) infusion.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CTX310 | Experimental | Drug: CTX310Subjects will receive an intravenous (IV) infusion. |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (6 listed)
- Age of ≥18 and ≤75 years at the time of signing the informed consent.
- Able to provide written informed consent.
- Participants diagnosed with persistent dyslipidemias defined by TG ≥150 mg/dL - and LDL-C ≥70 mg/dL in participants with ASCVD, or LDL-C ≥70 or 100mg/dL in participants with or without ASCVD respectively, or TG ≥500 mg/dL.
- Refractory to the maximal intensity or MTD of standard of care lines of lipid-lowering therapies available through routine clinical care, for at least 12 weeks prior to screening
- Female participants must be postmenopausal or surgically sterile.
Key exclusion criteria (11 listed)
- Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply.
- Evidence of liver disease, defined as but not limited to:
- LFTS >2 × upper limit of normal (ULN), or total bilirubin >2 × ULN, or INR >1.5 × ULN, or liver stiffness measured by liver elastography
- Abnormal or compromised function of kidney, heart, blood or liver.
- Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1.
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 |
|---|---|---|
| CTX310CTX310 is a lipid nanoparticle (LNP) formulation of clustered regularly interspaced short palindromic repeats (CRISPR)-associated protein 9 (Cas9) components for in vivo editing of the target gene angiopoietin-like 3 (ANGPTL3). | Drug | CTX310 |
Primary outcome
To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments
Time frame: From CTX310 infusion up to 12 months
Incidence of dose-limiting toxicities and frequency of adverse events
4 secondary outcomes
To assess the preliminary efficacy of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Time frame: Over 12 months, compared to baseline
To further characterize the safety of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Time frame: From CTX310 infusion up to 12 months
To assess the pharmacokinetics (PK) of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Time frame: From CTX310 infusion up to 12 months
To assess the pharmacodynamic (PD) response of CTX310 in adult participants with dyslipidemias that are refractory to available treatments
Time frame: Over 12 months, compared to baseline
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 18 Sep 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.
17 sites in 4 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing CTX310 in people with Cardiovascular and Metabolic Disease, run by CRISPR Therapeutics AG at 17 sites in 4 countries.
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 6 inclusion and 11 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: looking for participants now, as of the sponsor's last update on 18 Sep 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 17 sites in United States, Australia, New Zealand, United Kingdom. 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. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this 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.
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 Clinical Trials (877-214-4634, medicalaffairs@crisprtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
To evaluate the safety of CTX310 in adult subjects with dyslipidemias that are refractory to available treatments (time frame: From CTX310 infusion up to 12 months). 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 18 Sep 2026.