Long-term Follow-Up for RGX-121

Regenxbio

Status

Invitation-only

Conditions

Mucopolysaccharidosis II

Treatments

Other: Long-term Follow-Up

Study type

Observational

Funder types

Industry

Identifiers

NCT04597385

RGX-121-5101

Details and patient eligibility

About

RGX-121-5101 is the long-term follow-up study to the RGX-121-101 first in human study where participants received RGX-121, a gene therapy intended to deliver a functional copy of the iduronate-2-sulfatase gene (IDS) to the central nervous system. This study will evaluate the long-term safety and efficacy of RGX-121.

Full description

This is a prospective follow-up study to evaluate the long-term safety and efficacy after a single administration of RGX-121. Eligible participants are those who previously have enrolled in clinical study RGX-121-101 and received a single intracisternal (IC) or intracerebroventricular (ICV) infusion of RGX-121. Enrollment of each participant will occur the same day or after the participant has completed the end of study (EOS) visit or early termination visit (ET) from the previous (parent) study. Participants will be followed in this study cumulatively for up to 5 years after RGX-121 administration (inclusive of the parent study) or until RGX-121 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 they enroll in the current study following RGX-121 administration in the parent study.

Enrollment

12 estimated patients

Sex

Male

Ages

28+ months old

Volunteers

No Healthy Volunteers

Inclusion criteria

To be eligible, a participant must have previously received RGX-121 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