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Gene therapy fails to improve vision in advanced retinitis pigmentosa trial

NCT ID NCT02556736

Disease control Sponsor: AbbVie 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
Running, but no longer taking on new participants.
Completed This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 completed Phase 1/2a trial tested a gene therapy called RST-001 (AGN-151597) in 14 people with advanced retinitis pigmentosa, a genetic eye disease that causes severe vision loss. All participants had very poor vision (hand motion or worse) at the start. The study aimed to check safety and tolerability, but the treatment did not show any improvement in vision. No serious related side effects were reported, but the lack of efficacy means this approach did not help these patients.

What this could mean

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

Active substance
AGN-151597 (gene therapy)
What this could lead to
If it had worked, this gene therapy could have pointed toward a treatment to slow vision loss in retinitis pigmentosa.
What could go wrong
The trial did not show improvement in vision. It was very early-stage (Phase 1/2a) with only 14 participants, all with severe vision loss, so results may not apply to others.

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 1/2

Runs two stages together: safety and dose first, then whether the treatment works.

Participants

14 people

The number who actually took part.

Started

Dec 2015

Finished

Oct 2024

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

18 years and older

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: Participants must meet all of the following criteria. 1. Age \>= 18 years. 2. Signed and dated written informed consent obtained from the patient. 3. Ability to comply with testing and all protocol tests. Exclusion Criteria: Any one of the following will exclude patients from being enrolled into the study: 1. Unable or unwilling to meet requirements of the study. 2. Participation in a clinical study (ocular or non-ocular) with an investigational drug, agent or therapy in the past six months.

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Conditions

The condition(s) this trial relates to.

retinitis pigmentosa

As listed by the trial registrant

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

Advanced retinitis pigmentosa

Contacts and locations

Locations

  • Cincinnati Eye Institute- Edgewood /ID# 236713

    Edgewood, Kentucky, 41017-3415, United States

  • Duke Eye Center /ID# 235715

    Durham, North Carolina, 27705, United States

  • Retina Foundation of the Southwest /ID# 235199

    Dallas, Texas, 75231, United States

  • University of California San Francisco - Mission Bay /ID# 235717

    San Francisco, California, 94158, United States