Testing a new gene editing treatment for patients with high cholesterol

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This Phase 1b study evaluates the safety of VERVE-201, a gene editing therapy designed to inactivate the ANGPTL3 gene in the liver, in patients suffering from refractory hypercholesterolemia. The study employs a single-ascending dose approach to assess the pharmacodynamic profile and safety of the treatment. Participants will receive VERVE-201 to determine its effectiveness in lowering low-density lipoprotein cholesterol (LDL-C) levels. The study aims to provide insights into the potential of gene editing as a therapeutic option for this condition.

Why it matters

Who can join: the full rules

Inclusion Criteria:

* Refractory hypercholesterolemia
* Refractory hypertriglyceridemia

Exclusion Criteria:

* Active or history of chronic liver disease
* Current treatment with monoclonal antibody targeting ANGPTL3 or prior treatment within specified timeframe
* Clinically significant or abnormal laboratory values as defined by the protocol

Where it's running

• Clinical Study Center
  Garden Grove, California, United States Recruiting
• Clinical Study Center
  Clearwater, Florida, United States Recruiting
• Clinical Study Center
  Miami Lakes, Florida, United States Recruiting
• Clinical Study Center
  Port Orange, Florida, United States Recruiting
• Clinical Study Center
  New York, New York, United States Recruiting
• Clinical Study Center
  Adelaide, Australia Recruiting

Study contacts

• Study coordinator: Trial questions or participation questions: 1-877-CTLILLY (1-877-285-4559) or
• Email: LillyTrials@Lilly.com
• Phone: 317-615-4559

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• VERVE-102 for adults with or at high risk for heart disease

  This study tests whether VERVE-102 can lower bad cholesterol (LDL-C) compared with a placebo in adults with plaque buildup or high risk for it.

  Recruiting Phase 2 Verve Therapeutics, Inc. · DeSoto, Texas · NCT07839936

• Enlicitide (MK-0616) for adults with high cholesterol in India

  This study tests whether enlicitide helps lower bad cholesterol (LDL-C) compared with placebo in adults with hyperlipidemia/hypercholesterolemia.

  Recruiting at 2 sites Phase 3 Merck Sharp & Dohme LLC · Belagavi, Karnataka and 1 other locations · NCT07821944

• Pemafibrate in people with high cholesterol not fully controlled by statins

  This study tests whether taking Pemafibrate at 0.2 mg or 0.4 mg daily for 12 weeks helps people with high cholesterol who still don’t respond well to statins, compared with a placebo.

  Recruiting at 10 sites Phase 4 Kowa Company, Ltd. · Hokkaido and 9 other locations · NCT07634484

• Ion775 for high triglyceride levels: safety, tolerability, and how well it works

  A drug called ION775 is being tested to see if it can safely lower fasting triglycerides in adults with hypertriglyceridemia or severe hypertriglyceridemia who are already on standard lipid medicines.

  Recruiting at 17 sites Phase 2 Ionis Pharmaceuticals, Inc. · Lincoln, California and 17 other locations · NCT07604974

• Dual PCSK9 Inhibition in secondary prevention for high LDL-C with statin intolerance

  This study tests whether combining inclisiran and alirocumab lowers LDL cholesterol better than either drug alone in people with ASCVD who can’t tolerate statins.

  Recruiting Phase 4 University Medical Centre Ljubljana · Ljubljana · NCT07581808

• Moderate-Intensity Statin + Ezetimibe vs High-Intensity Statin in CKD with ASCVD

  In adults with chronic kidney disease and established cardiovascular disease, this study tests whether a moderate-intensity statin plus ezetimibe can match the benefits of a high-intensity statin alone over three years.

  Recruiting Yonsei University · Seoul · NCT07524101

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.