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
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).
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- 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