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New study tracks daily life impact of rare eye disease

NCT ID NCT03295071

Knowledge-focused Sponsor: GenSight Biologics 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 study looked at 44 people with a rare inherited eye condition called Leber Hereditary Optic Neuropathy (LHON) that causes vision loss. Researchers reviewed medical records and asked participants about their vision, health, and quality of life. The goal was to better understand how the disease affects daily living and to gather information for future research.

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

44 people

The number who actually took part.

Started

Jan 2018

Finished

Jul 2020

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

The study will recruit at least 50 affected LHON subjects (both adult and pediatric) from global clinical sites at the following countries, but not limited to: Spain, Italy, France, United Kingdom, and the United States. Efforts will be done to maintain the population of at least 50 affected LHON subjects. Recruitment efforts of recruiting approximately 75% of eligible subjects with 11778/ND4 mutation and 30% of eligible subjects under the age of 18 at the time of index date, wherever possible, will be done at the study level.

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: * Subjects who have a confirmed and genotyped diagnosis of LHON; * Subjects with visual function outcomes data including at least 2 visual function assessments between 1 year and 3 years (+/- 4 weeks) after vision loss; * Subjects who are willing and able to provide written informed consent if required as per local regulations; * For LHON subjects under the age of 18 years, permission from a legal guardian to participate in the study; Exclusion Criteria: * Subjects who received any investigational drug, or participated in any LHON-related interventional clinical trial during the observational period; * Subjects without medical charts data available.

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Conditions

The condition(s) this trial relates to.

Eye Diseases, Hereditary eye disorder hereditary disease inborn mitochondrial metabolism disorder Leber hereditary optic neuropathy nervous system disorder neurodegenerative disease

As listed by the trial registrant

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

Leber hereditary optic neuropathy

Contacts and locations

Locations

  • Alkek Eye Center

    Houston, Texas, 77030, United States

  • CHNO Les Quinze Vingts

    Paris, 75012, France

  • CHU d'Angers

    Angers, 49100, France

  • Doheny Eye Center UCLA Pasadena

    Pasadena, California, 91105, United States

  • Emory University Hospital

    Atlanta, Georgia, 30322, United States

  • Institut Catala de Retina

    Barcelona, 08022, Spain

  • Massachusetts Eye and Ear Infirmary

    Boston, Massachusetts, 02114, United States

  • Moorfields Eye Hospital

    London, Greater London, EC1V 2PD, United Kingdom

  • Ospedale Bellaria

    Bologna, 40139, Italy

  • Ospedale San Raffaele

    Milan, 20132, Italy

  • Wills Eye Institute

    Philadelphia, Pennsylvania, 19107, United States

More trials for these conditions

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