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

Status unconfirmed

Conditions studied: Homozygous Familial Hypercholesterolemia (HoFH)

In brief

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.

Key facts

Study ID

NCT04080050

Run by

REGENXBIO Inc.

People needed

8

Starts

2019-09-30

Expected to finish

2025-09-29

Last updated by the study team

2023-03-09

Who can join

Age: 18 and older. Sex: any. Healthy volunteers: not accepted.

You may qualify if…

• To be eligible to participate in this study, a participant must have previously received RGX-501 in a separate parent trial, and the participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent after the nature of the study has been explained, prior to any research-related procedures.

You may not qualify if…

• None

Where it is running

• Columbus Location — Columbus, Ohio, United States
• Portland location — Portland, Oregon, United States
• Philadelphia location — Philadelphia, Pennsylvania, United States
• Nashville location — Nashville, Tennessee, United States
• Montreal location — Montreal, Quebec, Canada
• Rotterdam location — Rotterdam, Netherlands

Full record on ClinicalTrials.gov

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