Long Term Follow-Up for RGX-111

Regenxbio

Status

Invitation-only

Conditions

Mucopolysaccharidosis I

Treatments

Genetic: No Intervention

Study type

Observational

Funder types

Industry

Identifiers

NCT06103487

RGX-111-5101

Details and patient eligibility

About

RGX-111-5101 is a long-term follow up study that evaluates the long term safety 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.

Full description

This is a prospective, multicenter, observational, follow-up study to evaluate the long-term safety of RGX-111 after a single administration. Eligible participants are those who previously participated in a clinical study where they received a single IC or ICV administration of RGX-111. Enrollment of each participant in this study should occur on the same day or after the participant has completed the end of study (EOS) visit or early discontinuation visit (EDV) from a previous (parent) clinical study. Participants will be followed for up to five years after RGX-111 administration (inclusive of the parent study) or until RGX-111 is commercially available in the participant's country, whichever occurs first. No treatment will be directed under this observational protocol. The total study duration for each participant may vary depending on when s/he enrolls in the current study following RGX-111 administration in the parent study.

Enrollment

7 estimated patients

Sex

All

Volunteers

No Healthy Volunteers

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