Status:

UNKNOWN

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

Lead Sponsor:

REGENXBIO Inc.

Conditions:

Homozygous Familial Hypercholesterolemia (HoFH)

Eligibility:

All Genders

18+ years

Brief 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...

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 rec...

Eligibility Criteria

Inclusion

  • 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.

Exclusion

  • None

Key Trial Info

Start Date :

September 30 2019

Trial Type :

OBSERVATIONAL

Allocation :

ACTUAL

End Date :

September 29 2025

Estimated Enrollment :

8 Patients enrolled

Trial Details

Trial ID

NCT04080050

Start Date

September 30 2019

End Date

September 29 2025

Last Update

March 9 2023

Active Locations (6)

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Page 1 of 2 (6 locations)

1

Columbus Location

Columbus, Ohio, United States, 43210

2

Portland location

Portland, Oregon, United States, 97239

3

Philadelphia location

Philadelphia, Pennsylvania, United States, 19104

4

Nashville location

Nashville, Tennessee, United States, 37232