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Eye disease study lays groundwork for gene therapy breakthrough

NCT ID NCT04639635

Knowledge-focused Sponsor: Columbia University 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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 looks at a rare, inherited eye disease called CNGB1 retinitis pigmentosa that causes gradual vision loss and blindness. Researchers want to learn how the disease progresses by using eye exams and imaging tests over three years. The goal is to find the best ways to measure vision changes so that future gene therapy trials can be more effective. No treatment is given in this study.

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 20 people

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

Started

Mar 2019

Expected to finish

Feb 2027

An estimate. End dates often move.

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.

Who is studied

Patients are expected to present to the clinic at all age groups; enrollment of subjects \<18 years of age will be obtained by informed consent in the company of a parent or legal guardian. There will also be no gender-or ethnic/racial specific inclusion criteria. The enrollment of non-English speaking subjects is not expected.

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: * Diagnosis of CNGB1-associated RP by study physician, who are trained retinal specialists in the university clinic * Must be able to commit to 4 follow-up study visits (3 years)

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Conditions

The condition(s) this trial relates to.

retinitis pigmentosa retinitis pigmentosa 45

As listed by the trial registrant

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

Retinitis pigmentosa associated with CNGB1 mutations

How to take part

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

  1. The places running it

    6 sites in 4 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

  • Dr. Stephen H. Tsang

    New York, New York, 10032, United States

  • Eberhard Karls University Tubingen

    Tübingen, Germany

  • Institut de la Vision/Centre de maladies rares du Centre Hospitalier National Ophtalmologique des Quinze-Vingts

    Paris, France

  • Klinikum der Universität München University Eye Hospital, Ludwig-Maximilians-University (LMU) Munich

    München, Bavaria, 80336, Germany

  • Moorfields Eye Hospital NHS Foundation Trust

    London, United Kingdom

  • Wills Eye Hospital

    Philadelphia, Pennsylvania, 19107, United States