Actively Recruiting
A Phase 1 Open-label, Multicenter Trial Evaluating Safety and Tolerability of CTX310, a CRISPR-Cas9 Gene Editing Therapy for Refractory Dyslipidemias
Led by CRISPR Therapeutics AG · Updated on 2026-05-22
90
Participants Needed
18
Research Sites
52 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating CTX310, a lipid nanoparticle formulation of CRISPR-Guide RNA-Cas9 nuclease, in adults aged 18 to 75 with dyslipidemias that do not respond to available treatments. This phase 1, open-label, multicenter trial aims to assess the safety and tolerability of this in vivo gene editing approach targeting the ANGPTL3 gene. The study is sponsored by CRISPR Therapeutics AG and explores a new treatment option for difficult-to-treat lipid disorders.
CONDITIONS
Brief Title
A Safety and Tolerability Trial Evaluating CTX310 in Participants With Refractory Dyslipidemias
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Age between 18 and 75 years at the time of informed consent
- Able to provide written informed consent
- Diagnosed with persistent dyslipidemias defined by specific triglyceride and LDL-C levels as per study criteria
- Refractory to maximum tolerated or maximal intensity standard lipid-lowering therapies 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 use effective contraception during the study
You will not qualify if you...
- Diagnosis of familial chylomicronemia syndrome (some exceptions may apply)
- Evidence of liver disease including abnormal liver function tests or liver stiffness
- Abnormal or compromised kidney, heart, blood, or liver function
- Acute coronary syndrome or stroke within 24 weeks prior to Day 1
- Acute pancreatitis within 12 weeks prior to Day 1
- Use of hepatocyte-targeted small interfering RNA therapy within 365 days prior to Day 1 (except inclisiran)
- Positive serology for HIV, hepatitis B or C (except prior immunization)
- Any malignancy within the past 5 years except certain skin or cervical cancers
- Women of childbearing potential
- Other protocol-defined inclusion/exclusion criteria may apply per local regulations
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 12 months
Participants receive a dose of CTX310 via intravenous infusion as part of the study treatment.
Trial Site Locations
Total: 18 locations
1
Research Site 10
Jacksonville, Florida, United States, 32216
Not Yet Recruiting
2
Research Site 17
Orlando, Florida, United States, 32789
Actively Recruiting
3
Research Site 11
Port Orange, Florida, United States, 32127
Actively Recruiting
4
Research Site 14
Durham, North Carolina, United States, 27710
Not Yet Recruiting
5
Research Site 13
High Point, North Carolina, United States, 27260
Actively Recruiting
6
Research Site 9
Cleveland, Ohio, United States, 44195
Not Yet Recruiting
7
Research Site 12
Portland, Oregon, United States, 97239
Not Yet Recruiting
8
Research Site 1
Adelaide, Australia, 5000
Actively Recruiting
9
Research Site 15
Brisbane, Australia, 4064
Not Yet Recruiting
10
Research Site 16
Brisbane, Australia
Not Yet Recruiting
11
Research Site 7
Camperdown, Australia, 2050
Not Yet Recruiting
12
Research Site 3
Heidelberg, Australia, 3084
Actively Recruiting
13
Research Site 2
Melbourne, Australia, 3168
Actively Recruiting
14
Research Site 5
Auckland, New Zealand, 2025
Actively Recruiting
15
Research Site 4
Christchurch, New Zealand, 8011
Actively Recruiting
16
Research Site 18
Cambridge, United Kingdom
Not Yet Recruiting
17
Research Site 8
London, United Kingdom, E1 1BB
Not Yet Recruiting
18
Research Site 6
London, United Kingdom, SE1 1YR
Actively Recruiting
Research Team
C
Clinical Trials
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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