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

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

Status

Active, not recruiting

Phases

Phase 2

Study type

Observational

Source

NL-OMON

Registry ID

NL-OMON49424

Enrollment

Registered

2020-06-25

Start date

2020-11-27

Completion date

Unknown

Last updated

2025-09-01

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

familial hypercholesterolaemia

Interventions

None listed

Eligibility

Age

18 Years to 99 Years

Inclusion criteria

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

Exclusion criteria: see inclusion criteria

Design outcomes

Primary

Measure	Time frame
The primary objective is to evaluate the long-term safety of RGX-501.	—

Secondary

Measure	Time frame
Secondary objectives are * To evaluate the long-term effect of RGX-501 on LDL-C and other lipid parameters, and * To evaluate the long-term impact of RGX-501 on the use of other lipid-lowering therapies, including apheresis.	—

Countries

Netherlands

Outcome results