REALITY LHON Registry (REALITY)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03295071|
Recruitment Status : Recruiting
First Posted : September 27, 2017
Last Update Posted : May 7, 2018
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.
|Condition or disease||Intervention/treatment|
|Leber Hereditary Optic Neuropathy||Other: Patient-reported outcomes (PROs)|
|Study Type :||Observational [Patient Registry]|
|Estimated Enrollment :||50 participants|
|Target Follow-Up Duration:||3 Years|
|Official Title:||Observational Registry Study of Leber Hereditary Optic Neuropathy (LHON) Affected Patients|
|Actual Study Start Date :||January 3, 2018|
|Estimated Primary Completion Date :||March 2019|
|Estimated Study Completion Date :||March 2019|
Other: Patient-reported outcomes (PROs)
Patient-reported outcomes (PROs)
- Visual Function [ Time Frame: All assessments available before enrollment ]Visual function will include visual examination data from medical records.
- The National Eye Institute Visual Function Questionnaire (VFQ)-25 [ Time Frame: Enrollment ]The National Eye Institute Visual Function Questionnaire (NEI-VFQ or VFQ)-25 is a valid and reliable 25-item version of the 51-item VFQ.
- The 36-Item Short Form Health Survey (SF-36) [ Time Frame: Enrollment ]The SF-36 surveys health status and quality of life
- Child Health Questionnaire (CHQ) [ Time Frame: Enrollment ]The CHQ uses the same structure and methodological approach as the SF-36 and is designed and normed for children from 5-to-18 years of age.
- EuroQol-5 Dimension (EQ-5D)-5L [ Time Frame: Enrollment ]The EuroQol-5 Dimension-5 Level (EQ-5D-5L) is a generic HRQoL instrument that is widely used as a PRO measure.
- Economic burden of disease [ Time Frame: Enrollment ]Data will be gathered by questionnaire at cross-sectional survey. Direct costs and indirect costs resulting from LHON will be estimated by all enrolled subjects. Direct costs will include the medical and non-medical direct costs due to LHON
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03295071
|Contact: Barrett Katz, MD||+1 646 831 email@example.com|
|Contact: Serge Fitoussi, MD||+33 (0)6 65 75 78 firstname.lastname@example.org|
|United States, Pennsylvania|
|Wills Eye Institute||Recruiting|
|Philadelphia, Pennsylvania, United States, 19107|
|Contact: Maria Massini email@example.com|
|Principal Investigator: Mark Moster, MD|