The primary study objective is long-term safety of RGX-501 and secondarily evaluation of the long-term effects of RGX-501 on LDL-C and other lipid parameters.
ID
Source
Brief title
Condition
- Lipid metabolism disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The primary objective is to evaluate the long-term safety of RGX-501.
Secondary outcome
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.
Background summary
RGX-501 is an investigational gene therapy in patients with HoFH. RGX-501 is a
liver-directed AAV vector serotype 8 (AAV8) based gene therapy that contains
genes that allow expression of functional LDLR in transfected cells. It is
anticipated that, following gene therapy administration, hepatocytes will be
able to express functional, transgenic LDLRs that can bind to and remove LDL-C
from circulation.
Study objective
The primary study objective is long-term safety of RGX-501 and secondarily
evaluation of the long-term effects of RGX-501 on LDL-C and other lipid
parameters.
Study design
This is a prospective, observational study to evaluate the long-term safety and
efficacy after a single administration of RGX-501.
Study burden and risks
The possible risks with joining this study include the risk and discomfort from
blood draws. The risks of drawing blood include temporary discomfort from the
needle in your arm, fainting, bruising, clotting, and swelling at the needle
site and, in rare instances, infection.
Blackwell Road 9600 Suite 210
Maryland 20850
US
Blackwell Road 9600 Suite 210
Maryland 20850
US
Listed location countries
Age
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
Design
Recruitment
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| EudraCT | EUCTR2019-004496-39-NL |
| ClinicalTrials.gov | NCT04080050 |
| CCMO | NL73955.000.20 |