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Looking for participantsPhase1

A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias

Sponsor: CRISPR Therapeutics AG

NCT ID: NCT07491172

View on ClinicalTrials.gov ↗

At a glance

What the study gets you
Access to the study treatment being tested
Type of study
Interventional (receives a drug or procedure)
Time in hospital
In-person visits at study sites — visit count not specified by the sponsor
Drug or intervention
CTX310 (drug)
How long the study runs
Study runs about 48 months (dates as stated)
About the drug or intervention
CTX310 — drug: 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).
Patient visit burden
Not specified by the sponsor
Type of study
Testing a treatment
Ages
18 Years to 75 Years
Who
All
Number of participants
90
Started
2024-06-21
Last checked
2026-09

Plain English Summary

What is this study?

  • • Testing a new treatment for cardiovascular
  • • Phase1 - 90 participants
  • • 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

Who can take part?

  • • Ages 18 Years to 75 Years
  • • Diagnosed with cardiovascular

Where?

  • • Cambridge - Royal Papworth Hospital
  • • London - Richmond Pharmacology

This is a simplified summary. Always discuss with your doctor before making any decisions.

About This Trial

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.

More detail

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.

CardiovascularMetabolic DiseaseDyslipidemiasLipid DisorderHypertriglyceridemiaHeterozygous Familial Hypercholesterolemia (HeFH)Homozygous Familial Hypercholesterolemia (HoFH)Severe Hypertriglyceridemia (sHTG)Mixed HyperlipemiaHypercholesterolaemia

How this trial compares with your answers

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What we know so far

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Preliminary match based on your answers. Full eligibility requires on-site assessment including medical history, physical exam, and lab tests. This does not guarantee enrolment.

Eligibility at a Glance

Key info

  • Age: 18 Years - 75 Years
  • Who can join: All genders

What the study is looking for

  • ✓Age of ≥18 and ≤75 years at the time of signing the agreement to take part.
  • ✓Able to provide written agreement to take part.
  • ✓Refractory to the maximal intensity or MTD of the usual treatment lines of lipid-lowering therapies available through...
  • ✓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...

Who cannot take part

  • ✗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 liver blood test \>2 × ULN, or INR \>1.5 × ULN, or liver stiffness...
  • ✗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...
See the full criteria
Key Inclusion Criteria: 1. Age of ≥18 and ≤75 years at the time of signing the informed consent. 2. Able to provide written informed consent. 3. 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. 4. 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 5. Female participants must be postmenopausal or surgically sterile. 6. 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. Exclusion Criteria: 1. Participants with familial chylomicronemia syndrome (FCS). Some exceptions may apply. 2. 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 3. Abnormal or compromised function of kidney, heart, blood or liver. 4. Acute coronary syndrome event or stroke within 24 weeks prior to Day 1. Acute pancreatitis within 12 weeks prior to Day 1. 5. Current use or use within 365 days from Day 1 of any hepatocyte-targeted small interfering RNA (except inclisiran). 6. Positive serology for HIV, hepatitis B or hepatitis C (antibody, surface antigen orNAT). Serology consistent with prior immunization will be eligible for the trial. 7. 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). 8. 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.

Where Is This Study? (2 UK sites)

Royal Papworth Hospital

Cambridge, United Kingdom

Recruiting
Site contact (verified)

Richmond Pharmacology

London SE1 1YR, United Kingdom

Recruiting
Site contact (verified)

How to Get in Touch

Clinical Trials

Sponsor contact

CONTACT

877-214-4634 medicalaffairs@crisprtx.com
Data sourced from ClinicalTrials.gov · Last verified: 2026-09