NCT04080050

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501

• Status: Unknown
• Phase: —
• Study type: Observational
• Enrollment: 8 (actual)
• Sponsor: REGENXBIO Inc. · Industry
• Sex: All
• Age: 18 Years
• Healthy volunteers: Not accepted

Summary

This long-term observational study is designed to follow subjects who, during another Clinical Study, received gene therapy treatment used to treat their Homozygous Familial Hypercholesterolemia (HoFH) disease. This study is intended to follow those subjects for up to 5 years since they received treatment to look for any long-term safety concerns. There is no investigational drug or therapy provided as part of this study.

Detailed description

Homozygous Familial Hypercholesterolemia (HoFH) is a rare genetic metabolic disorder characterized by absent or severely reduced capacity to catabolize circulating LDL particles by the hepatic LDL receptor. As a consequence, HoFH subjects present abnormal total plasma cholesterol (LDL-C) levels, resulting in severe atherosclerosis often leading to early onset of cardiovascular disease. Early initiation of aggressive treatment for these patients is therefore essential. Unfortunately, despite existing therapies, treated LDL-C levels could remain well above acceptable levels. Thus, the functional replacement of the defective LDLR via AAV-based liver-directed gene therapy, RGX-501, may be a viable approach to treat this disease and improve response to current lipid-lowering treatments. This is a prospective, observational study to evaluate the long-term safety and efficacy after a single administration of RGX-501. Eligible participants are those who previously have enrolled in a clinical study and received a single intravenous infusion of RGX-501.

Conditions

• Homozygous Familial Hypercholesterolemia (HoFH)

Interventions

Type	Name	Description
DRUG	Single intravenous (IV) dose of human Low Density Lipoprotein Receptor (LDLR) Gene Therapy	No investigational product will be administered in this study. All participants have previously received a one-time intravenous infusion of RGX-501 in a separate clinical trial

Timeline

• Start date: 2019-09-30
• Primary completion: 2024-09-29
• Completion: 2025-09-29
• First posted: 2019-09-06
• Last updated: 2023-03-09

Locations

6 sites across 3 countries: United States, Canada, Netherlands

Regulatory

• FDA-regulated drug study