Long Term Follow-Up for RGX-111

Conditions

• Mucopolysaccharidosis I

Eligibility Criteria

Sex

ALL

Age

N/A - N/A

Healthy Volunteers

Not accepted

Interventions

• No Intervention — GENETIC
  Observational study

Study Details

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.

Key Dates

First listed

Oct 26, 2023

Start date

Jul 24, 2023

Status verified

Apr 2026

Primary completion

Sep 30, 2027

Completion

Sep 30, 2027

Study Design

Enrollment

21 participants (estimated)

Arms

• Arm: RGX-111 Recipients
  Subjects who have received RGX-111 in a separate parent study.

Primary Outcome Measure

Evaluation of the long-term safety of RGX-111 [ Time Frame: 5 years inclusive of parent study ]

Locations (1)

Find similar trials in Orange, CA

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