Clinical trial

A Long-term Follow-up Study to Evaluate the Safety and Efficacy of RGX-111

Name

RGX-111-5101

Description

RGX-111-5101 is a long-term follow up study that evaluates the long term safety and efficacy of RGX-111 in participants who have received RGX-111 (a gene therapy intended to deliver a functional copy of the alpha-L-iduronidase gene (IDUA) to the central nervous system) in a separate parent study.

Trial arms

Trial start

2023-07-24

Estimated PCD

2027-09-01

Trial end

2027-09-01

Treatment

No Intervention

Observational study

Arms:

RGX-111 Recipients

Size

Primary endpoint

Evaluation of the long-term safety of RGX-111

5 years inclusive of parent study

Eligibility criteria

Inclusion Criteria: * To be eligible, a participant must have previously received RGX-111 in a separate parent trial. * Participant or participant's legal guardian(s) is/(are) willing and able to provide written, signed informed consent. Exclusion Criteria: * None.

Protocol

{'studyType': 'OBSERVATIONAL', 'patientRegistry': False, 'designInfo': {'observationalModel': 'OTHER', 'timePerspective': 'PROSPECTIVE'}, 'enrollmentInfo': {'count': 8, 'type': 'ESTIMATED'}}

Updated at

2023-10-26

1 organization

1 product

1 indication

Organization

REGENXBIO

Product

No Intervention

Indication

Mucopolysaccharidosis Type I