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Gene therapy watch: RGX-111 safety tracked in MPS i patients

NCT ID NCT06103487

Disease control Sponsor: REGENXBIO Inc. Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only This study
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 27, 2026 · Last updated Jun 27, 2026

Summary

This study checks the long-term safety of RGX-111, a gene therapy for people with MPS I (a rare genetic disorder). It follows 21 participants who already received the therapy in an earlier trial. Researchers will monitor side effects and measure changes in thinking and behavior over time.

Why investors are watching

RegenxBio is testing RGX-111, a gene therapy that delivers a working copy of the IDUA gene to the brain, in a long-term follow-up of 21 patients who already received the treatment in an earlier study. For a small company, this readout matters because it will show whether the therapy stays safe and keeps working over time, which is the key question for a one-time gene therapy. A clear safety record could support the therapy's path toward approval, while safety problems could set the program back.

If it works: If the follow-up shows RGX-111 remains safe and continues to control the disease, RegenxBio could move closer to seeking regulatory approval for the therapy. That would give the company a potential product in a rare disease area with few treatment options.

If it fails: If the follow-up reveals new safety issues or shows the therapy's effect fades, RegenxBio would face a major setback, as the company's value depends heavily on this program. Clinical trials often fail or face delays, and a small company has limited resources to absorb such a blow.

AI-written from the trial record. Speculative, and not investment advice.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Participants

About 21 people

The number the study aims to enrol. It can still change while the study runs.

Started

Jul 2023

Expected to finish

Sep 2027

An estimate. End dates often move.

Lead sponsor

A company

The lead sponsor is a pharmaceutical, biotech, or medical-device company.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Who is studied

Received RGX-111 in a separate parent trial.

Ages

Children (under 18), adults (18 to 64) and older adults (65 and over)

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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.

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Conditions

The condition(s) this trial relates to.

Mucopolysaccharidosis I mucopolysaccharidosis type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Mucopolysaccharidosis I

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    2 sites in 2 countries. The list below names each one and where it is.

  2. The official record

    The full official record for this study. This one lists no contact details, but it is the first place any would appear.

    Open the record ↗

  3. A doctor treating you

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's Hospital of Orange County

    Orange, California, 92868, United States

  • Hospital de Clinicas de Porto Alegre

    Porto Alegre, Rio Grande do Sul, 90035-903, Brazil

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