Cure My Disease

Don't miss your cure!

 Log in / Sign up

Mapping the slow fade of RPE65 blindness to speed future cures

NCT ID NCT02714816

NEW Knowledge-focused Sponsor: MeiraGTx UK II Ltd 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 Sep 21, 2026 · Last updated Sep 21, 2026

Summary

Researchers are following people with Leber congenital amaurosis caused by RPE65 gene mutations to see how their vision and retinas change over time. The study enrolls children and adults, ages 3 and older, who have this inherited retinal dystrophy. Participants undergo regular eye exams, imaging, and questionnaires, but receive no experimental treatment. The goal is to build a detailed picture of the disease's natural course, which could help future trials measure whether new therapies work.

What this could mean

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

What this could lead to
Detailed maps of how RPE65-related blindness progresses could help researchers design and measure future gene therapies more effectively.
What could go wrong
This study only observes participants and gives no treatment, so it cannot restore vision. Its small size and focus on one rare genetic form of blindness may limit how far the results apply.

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

37 people

The number who actually took part.

Start date

Apr 2016

Finished

Jul 2023

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

Patients with RPE65-LCA condition

Ages

3 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: * Patients with RPE65 associated retinal dystrophy * Minimum subject age of 3 years * Able to give consent/parent or guardian able to give consent Exclusion Criteria: * Patients unable or unwilling to undertake consent or clinical testing * Have received a gene therapy treatment in both eyes

Get updates

Get notified about this study

Sign up to get updates when this study changes or when new studies for Leber congenital amaurosis are added.

Our safety recommendation!

By submitting, you agree to our Terms of use

Conditions

The condition(s) this trial relates to.

Leber congenital amaurosis Leber congenital amaurosis 2

As listed by the trial registrant

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

Leber congenital amaurosis

Contacts and locations

Locations

  • Kellogg Eye Center

    Ann Arbor, Michigan, MI 48105, United States

  • Moorfields Eye Hospital

    London, United Kingdom

More trials for these conditions

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