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Gene Therapy's lasting effects on vision tracked in Long-Term study

NCT ID NCT03584165

Disease control Sponsor: NightstaRx Ltd, a Biogen Company Source: ClinicalTrials.gov ↗

What the study statuses mean

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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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Jul 06, 2026 · Last updated Jul 07, 2026 · Updated 1 time

Summary

This study follows people with two inherited eye conditions—choroideremia and X-linked retinitis pigmentosa—who previously received gene therapy. Researchers monitor their safety and vision over several years to see if the treatment remains safe and helps preserve sight. Participants include both those who got the gene therapy and untreated controls from earlier studies.

What this could mean

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

Active substance
gene therapy (BIIB111 or BIIB112)
What this could lead to
If successful, this could show that a single gene therapy treatment provides lasting safety and helps preserve vision for years in people with these inherited eye diseases.
What could go wrong
This is a follow-up study, not a new treatment test. It cannot prove the therapy works for everyone, and long-term side effects or loss of vision are still possible.

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 3

Large-scale testing in a bigger group. Usually the last step before a treatment can be approved.

Participants

277 people

The number who actually took part.

Started

Jun 2018

Finished

Jun 2026

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

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

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.

Key Inclusion Criteria: CHM Participants a. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of BIIB111 for CHM. XLRP Participants a. Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study. Key Exclusion Criteria: Participants are not eligible for study participation if they meet the following exclusion criterion. a. In the opinion of the Investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study. NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

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Conditions

The condition(s) this trial relates to.

choroideremia retinitis pigmentosa retinitis pigmentosa 3

As listed by the trial registrant

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

Choroideremia X-linked retinitis pigmentosa

Contacts and locations

Locations

  • CHU Montpellier - Saint Eloi

    Montpellier, 34295, France

  • Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts

    Paris, 75012, France

  • Cincinnati Eye Institute - Blue Ash

    Cincinnati, Ohio, 45242-5664, United States

  • Columbia University Medical Center

    New York, New York, 10032, United States

  • Helsinki University Central Hospital (HUCH)

    Helsinki, 00290 HUS, Finland

  • Instituto Genetica Ocular

    São Paulo, 04039, Brazil

  • John Radcliffe Hospital

    Oxford, OX3 9DU, United Kingdom

  • Johns Hopkins Hospital

    Baltimore, Maryland, 21287-0005, United States

  • MEEI Massachusets Eye and Ear Infirmary

    Boston, Massachusetts, 02114-3002, United States

  • Manchester Royal Eye Hopsital

    Manchester, M13 9WL, United Kingdom

  • McGill University Health Centre

    Montreal, Quebec, H4A 3JI, Canada

  • Moorfields Eye Hospital

    London, EC1V 2PD, United Kingdom

  • OHSU - Casey Eye Institute

    Portland, Oregon, 97239, United States

  • Radboudumc

    Nijmegen, 6525 GA, Netherlands

  • Retina Foundation of the Southwest

    Dallas, Texas, 75231-5080, United States

  • Rigshospitalet-Glostrup, Oejenafdelingen

    Glostrup Municipality, 2600, Denmark

  • Southampton General Hospital

    Southampton, SO16 6YD, United Kingdom

  • The Northern Alberta Clinical Trials and Research Centre

    Edmonton, Alberta, T5H 3V9, Canada

  • The University of British Columbia - Eye Care Centre

    Vancouver, British Columbia, V5Z 39N, Canada

  • UCLA - Jules Stein Eye Institute

    Los Angeles, California, 90095-7065, United States

  • Universitats Klinikum Tubingen - Institute for Ophthalmic Research

    Tübingen, 72076, Germany

  • University of Miami

    Miami, Florida, 33136, United States

  • University of Wisconsin School of Medicine

    Madison, Wisconsin, 53705-3644, United States

  • Universitäts-Augenklinik Bonn

    Bonn, 53127, Germany

  • Vitreo Retinal Associates PA - The Millennium Center

    Gainesville, Florida, 32607, United States

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