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

Phase 1

• Conditions: Adults with homozygous familial hypercholesterolemia; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2019-004496-39-NL
• Lead Sponsor: REGENXBIO Inc.

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2020-11-13
• Last Update Posted: 16 November 2020

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 12

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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 12
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

Exclusion Criteria

None

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified