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

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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