Leber Hereditary Optic Neuropathy Clinical Trial
— REALITYOfficial title:
Observational Registry Study of Leber Hereditary Optic Neuropathy (LHON) Affected Patients
Verified date | July 2020 |
Source | GenSight Biologics |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Observational [Patient Registry] |
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).
Status | Completed |
Enrollment | 44 |
Est. completion date | July 1, 2020 |
Est. primary completion date | July 1, 2020 |
Accepts healthy volunteers | No |
Gender | All |
Age group | N/A and older |
Eligibility | 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. |
Country | Name | City | State |
---|---|---|---|
France | CHU d'Angers | Angers | |
France | CHNO Les Quinze Vingts | Paris | |
Italy | Ospedale Bellaria | Bologna | |
Italy | Ospedale San Raffaele | Milano | |
Spain | Institut Catala de Retina | Barcelona | |
United Kingdom | Moorfields Eye Hospital | London | Greater London |
United States | Emory University Hospital | Atlanta | Georgia |
United States | Massachusetts Eye and Ear Infirmary | Boston | Massachusetts |
United States | Alkek Eye Center | Houston | Texas |
United States | Doheny Eye Center UCLA Pasadena | Pasadena | California |
United States | Wills Eye Institute | Philadelphia | Pennsylvania |
Lead Sponsor | Collaborator |
---|---|
GenSight Biologics |
United States, France, Italy, Spain, United Kingdom,
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | Visual Function | Visual function will include visual examination data from medical records. | All assessments available before enrollment | |
Primary | The National Eye Institute Visual Function Questionnaire (VFQ)-25 | 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. | Enrollment | |
Primary | The 36-Item Short Form Health Survey (SF-36) | The SF-36 surveys health status and quality of life | Enrollment | |
Primary | Child Health Questionnaire (CHQ) | 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. | Enrollment | |
Primary | EuroQol-5 Dimension (EQ-5D)-5L | The EuroQol-5 Dimension-5 Level (EQ-5D-5L) is a generic HRQoL instrument that is widely used as a PRO measure. | Enrollment | |
Secondary | Economic burden of disease | 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 | Enrollment |
Status | Clinical Trial | Phase | |
---|---|---|---|
Recruiting |
NCT03475173 -
New Non-invasive Modalities for Assessing Retinal Structure and Function
|
N/A | |
Active, not recruiting |
NCT03293524 -
Efficacy & Safety Study of Bilateral IVT Injection of GS010 in LHON Subjects Due to the ND4 Mutation for up to 1 Year
|
Phase 3 | |
Recruiting |
NCT02176733 -
Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy
|
Phase 2 | |
Completed |
NCT03406104 -
RESCUE and REVERSE Long-term Follow-up
|
Phase 3 | |
Recruiting |
NCT03011541 -
Stem Cell Ophthalmology Treatment Study II
|
N/A | |
Completed |
NCT02064569 -
Safety Evaluation of Gene Therapy in Leber Hereditary Optic Neuropathy (LHON) Patients
|
Phase 1/Phase 2 | |
Active, not recruiting |
NCT03153293 -
A Single Intravitreal Injection of rAAV2-ND4 for the Treatment of Leber's Hereditary Optic Neuropathy
|
Phase 2/Phase 3 | |
Completed |
NCT01267422 -
Safety and Efficacy Study of rAAV2-ND4 Treatment of Leber Hereditary Optic Neuropathy (LHON)
|
N/A |