CompletedNot Applicable

REALITY LHON Registry

United States, France, Italy44 participantsStarted 2018-01-03

Plain-language summary

This study is a multi-country retrospective and cross-sectional observational study of affected LHON subjects, based on retrospective subjects' medical chart abstractions and cross-sectional administration of patient-reported outcomes (PROs).

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

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.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Visual Function

Timeframe: All assessments available before enrollment

The National Eye Institute Visual Function Questionnaire (VFQ)-25

Timeframe: Enrollment

The 36-Item Short Form Health Survey (SF-36)

Timeframe: Enrollment

Child Health Questionnaire (CHQ)

Timeframe: Enrollment

EuroQol-5 Dimension (EQ-5D)-5L

Timeframe: Enrollment

Trial details

NCT IDNCT03295071

SponsorGenSight Biologics

Sponsor typeINDUSTRY

Study typeOBSERVATIONAL

Primary completion2020-07-01

View on ClinicalTrials.gov More Leber Hereditary Optic Neuropathy trials

Who can participate

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

What they're measuring

Visual Function

Timeframe: All assessments available before enrollment

The National Eye Institute Visual Function Questionnaire (VFQ)-25

Timeframe: Enrollment

The 36-Item Short Form Health Survey (SF-36)

Timeframe: Enrollment

Child Health Questionnaire (CHQ)

Timeframe: Enrollment

EuroQol-5 Dimension (EQ-5D)-5L

Timeframe: Enrollment