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Gene therapy targets second eye in rare blindness disorder

NCT ID NCT06646289

Disease control Sponsor: Janssen Research & Development, LLC 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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Aug 28, 2026 · Updated 3 times

Summary

This study tests a gene therapy called botaretigene sparoparvovec in the second eye of people with X-linked retinitis pigmentosa, a genetic condition that causes vision loss. The 24 participants previously received the therapy in one eye and are now being followed for safety and vision changes in the other eye. The goal is to see if treating both eyes is safe and may help preserve or improve sight.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
botaretigene sparoparvovec (gene therapy)
What this could lead to
If successful, this could improve vision in the second eye for people with X-linked retinitis pigmentosa, a genetic cause of blindness.
What could go wrong
This is a small, early-phase study (24 participants) focused on safety, so it may not lead to a proven treatment. Gene therapy carries risks like inflammation or vision changes.

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.

Phase

Phase 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

24 people

The number who actually took part.

Started

Oct 2024

Expected to finish

Oct 2030

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.

Ages

5 years and older

Sex

Male participants only

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: * Have been treated with AAV5-hRKp.RPGR in study MGT009 and have completed or is currently enrolled in Study MGT010 * Must sign an informed consent form indicating that they understand the purpose and procedures of the study and is willing to participate in the study * Willing to adhere to the protocol and long-term follow-up Exclusion Criteria: \- There are no specific exclusion criteria to enroll in this study

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Conditions

The condition(s) this trial relates to.

retinitis pigmentosa retinitis pigmentosa 3

As listed by the trial registrant

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

X-linked retinitis pigmentosa

Contacts and locations

Locations

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114, United States

  • Moorfields Eye Hospital

    London, EC1V 2PD, United Kingdom

  • University of Michigan Kellogg Eye Center

    Ann Arbor, Michigan, 48105, United States

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Other studies related to the condition(s) this trial covers.