Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)

Completed

• Conditions: Leber's Hereditary Optic Neuropathy (LHON)

• Registration Number: NCT02796274
• Lead Sponsor: Santhera Pharmaceuticals

Brief Summary

The purpose of this survey is to collect visual acuity data from patients with LHON in order to establish the clinical course (natural history) and visual acuity outcomes in patients with a genetically confirmed diagnosis of LHON. In addition, this survey will generate data that will serve as comparator for the open-label study SNT-IV-006.

Detailed Description

Not available

Study Timeline

• Study Start: 2016-05
• Study First Submitted: 2016-06-07
• Study First Submitted QC: 2016-06-07
• Study First Posted: 2016-06-10
• Primary Completion: 2018-03
• Study Completion: 2018-07
• Results First Submitted: 2019-10-01
• Status Verified: 2020-01
• Results First Submitted QC: 2020-01-20
• Last Update Submitted: 2020-01-20
• Results First Posted: 2020-01-30
• Last Update Posted: 2020-01-30

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 219

Inclusion Criteria

1. age ≥ 12 years
2. the onset of symptoms is dated after 1999 and is well documented (at least month of onset of symptoms is known for each eye)
3. at least two VA assessments are available within 5 years of onset of symptoms and prior to idebenone use
4. have a genetic diagnosis for LHON for one of the following mitochondrial DNA (mtDNA) mutations: G11778A, G3460A, T14484C

Exclusion Criteria

1. any participation in an interventional clinical trial after the onset of symptoms
2. any other cause of visual impairment (e.g. glaucoma, diabetic retinopathy, AIDS related visual impairment, cataract, macular degeneration, etc.) or any active ocular disorder (uveitis, infections, inflammatory retinal disease, thyroid eye disease, etc.) during the data collection period

Study & Design

• Study Type: OBSERVATIONAL
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
In Eyes With VA Assessment Made ≤1 Year After Onset of Symptoms: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA (Measured by Change in ETDRS Letters) From Baseline (BL) or in Which BL VA Better Than 1.0 logMAR Was Maintained at 12 Months	12 months	Clinically Relevant Recovery (CRR) is defined as a VA improvement from off-chart (ETDRS) to 5 letters on-chart, or an on-chart improvement of 10 letters.

Trial Locations

Locations (21)

Universitair Ziekenhuis Gent; 🇧🇪 Gent, Belgium

C. H. U. Sart Tilman; 🇧🇪 Liege, Belgium

CHU Angers - Hôpital Hôtel Dieu; 🇫🇷 Angers, France

Hopital Roger Salengro - CHU Lille; 🇫🇷 Lille, France

Fondation Ophtalmologique Adolphe de Rothschild; 🇫🇷 Paris, France

CHU Reims - Hôpital Robert Debré; 🇫🇷 Reims, France

CHU Strasbourg - Hôpital Hautepierre; 🇫🇷 Strasbourg, France

Justus-Liebig-Universitaet Giessen; 🇩🇪 Giessen, Germany

Universitaetsklinikum Heidelberg; 🇩🇪 Heidelberg, Germany

Friedrich-Baur-Institut; 🇩🇪 Muenchen, Germany

Fondazione IRCCS Istituto Neurologico Carlo Besta; 🇮🇹 Milano, Italy

Azienda Ospedaliera Universitaria Policlinico G. Martino; 🇮🇹 Messina, Italy

Maastricht University Medical Center; 🇳🇱 Maastricht, Netherlands

Università di Pisa; 🇮🇹 Pisa, Italy

G. B. Bietti Fondazione - IRCCS; 🇮🇹 Rome, Italy

Haukeland Universitetssykehus; 🇳🇴 Bergen, Norway

University Hospital of Wales; 🇬🇧 Cardiff, United Kingdom

Queen's Hospital; 🇬🇧 London, United Kingdom

CHU Saint-Pierre; 🇧🇪 Brussels, Belgium

Oogziekenhuis Rotterdam; 🇳🇱 Rotterdam, Netherlands

Cliniques Universitaire Saint-Luc; 🇧🇪 Brussel, Belgium