A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-501
Phase 2
Completed
- Conditions
- 10013317familial hypercholesterolaemia
- Registration Number
- NL-OMON49424
- Lead Sponsor
- REGENXBIO Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Completed
- Sex
- Not specified
- Target Recruitment
- 2
Inclusion Criteria
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 Criteria
see inclusion criteria
Study & Design
- Study Type
- Observational invasive
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method <p>The primary objective is to evaluate the long-term safety of RGX-501.</p><br>
- Secondary Outcome Measures
Name Time Method <p>Secondary objectives are<br /><br>* To evaluate the long-term effect of RGX-501 on LDL-C and other lipid<br /><br>parameters,<br /><br>and<br /><br>* To evaluate the long-term impact of RGX-501 on the use of other lipid-lowering<br /><br>therapies, including apheresis.</p><br>