REALITY LHON Registry

GenSight Biologics

Status: Completed

Conditions: Leber Hereditary Optic Neuropathy

Treatments: Other: Patient-reported outcomes (PROs)

Study type: Observational

Funder types: Industry

Identifiers

NCT03295071

GS010_Registry_001

Details and patient eligibility

About

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).

Full description

The purpose of this study is to understand the evolution of visual functional and structural changes and other associated symptoms in patients with LHON. The relation between genetic, lifestyle and/or environmental factors and the LHON disease will be described, and a better understanding of the natural history of LHON disease and the healthcare associated to with the disease will be sought.

In addition, we would also like to understand the economic burden for patients and their families with LHON resulting from direct and indirect costs they may have because of their disease.

Enrollment: 44 patients

Sex: All

Volunteers: No Healthy Volunteers

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.

Trial design

44 participants in 1 patient group

Single-group study

Description:

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).

Treatment:

Other: Patient-reported outcomes (PROs)