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
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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.
1Since the REALITY LHON Registry has already completed enrollment and was collecting real-world data on how Leber Hereditary Optic Neuropathy affects vision and quality of life over time, can you tell me what findings or published results have come out of it that might be relevant to my situation?
2This registry measured quality of life using tools like the VFQ-25 and SF-36 — what do those scores typically look like for people at my stage of LHON, and how might they help us track how my condition is progressing?
3Because this was an observational registry rather than a treatment trial, it wasn't testing a new therapy — so what active treatment trials or approved options should I be looking at alongside this kind of data?
4The registry included questionnaires designed for children as well as adults, so if my family member with LHON is a child, are there specific findings from pediatric patients in this registry that could help us understand what to expect for them?
5Since this registry focused on tracking natural disease progression and patient-reported outcomes rather than testing an intervention, how does the data collected here help inform the treatment decisions my care team would make for me going forward?
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
1
Visual Function
Timeframe: All assessments available before enrollment
2
The National Eye Institute Visual Function Questionnaire (VFQ)-25