Heterozygous Familial Hypercholesterolemia research hub
3 indexed studies · 2 currently recruiting
- recruitingPHASE1
A Study of VERVE-102 in Patients With Familial Hypercholesterolemia or Premature Coronary Artery Disease
This study is testing a new gene-editing treatment called VERVE-102 for people with high cholesterol due to a genetic condition (Familial Hypercholesterolemia) or early heart disease. It aims to see if VERVE-102 is safe and can help lower 'bad' cholesterol.
United States · Australia · Canada - authorisedTherapeutic confirmatory (Phase III)
Randomized, Placebo-Controlled, Double-Blind, Phase 3b Study to Evaluate the Efficacy and Safety of Lerodalcibep in Children and Adolescents, 6 to 17 Years of Age, with Heterozygous Familial Hypercholesterolemia on Stable Diet and Oral Lipid-Lowering Therapy (LIBerate-Kids)
This study looks at a new medicine called lerodalcibep for children and teenagers aged 6 to 17 with a genetic condition causing high cholesterol (Heterozygous Familial Hypercholesterolemia). It aims to see if the medicine can lower their cholesterol safely and effectively, compared to a dummy medicine.
Norway - enrolling by invitation
Long-term Follow-up of Participants Dosed With an Investigational Gene Editing Therapy for Cardiovascular Disease
This study is following people who've had an experimental gene therapy for certain heart conditions. It checks how the treatment affects them over a long time, up to 15 years, looking for any lasting benefits or side effects to make sure it's safe and works well.
New Zealand · United Kingdom