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Gene Therapy

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

N/A
Waitlist Available
Research Sponsored by Regenxbio Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
* 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.
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up up to 5 years after receiving treatment with rgx-501
Awards & highlights
No Placebo-Only Group

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.

Eligible Conditions
  • Hypercholesterolemia

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~up to 5 years after receiving treatment with rgx-501
This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years after receiving treatment with rgx-501 for reporting.

Treatment Details

Study Objectives

Study objectives can provide a clearer picture of what you can expect from a treatment.
Primary study objectives
Number of incidents of new and unexpected adverse events and serious adverse events.
Secondary study objectives
Cocaine
The absolute LDL-C level in mg/dL by beta quantification
Therapeutic procedure

Awards & Highlights

No Placebo-Only Group
All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups
Experimental Treatment
Group I: RGX-501Experimental Treatment1 Intervention
Study participants who have received RGX-501 gene therapy in a separate parent trial

Find a Location

Who is running the clinical trial?

Regenxbio Inc.Lead Sponsor
19 Previous Clinical Trials
2,480 Total Patients Enrolled
2 Trials studying Hypercholesterolemia
13 Patients Enrolled for Hypercholesterolemia
REGENXBIO, Inc.Lead Sponsor
19 Previous Clinical Trials
2,480 Total Patients Enrolled
2 Trials studying Hypercholesterolemia
13 Patients Enrolled for Hypercholesterolemia
REGENXBIO Inc.Lead Sponsor
24 Previous Clinical Trials
2,737 Total Patients Enrolled
2 Trials studying Hypercholesterolemia
13 Patients Enrolled for Hypercholesterolemia
~1 spots leftby Dec 2025
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