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

NCT ID: NCT02796274

Last Updated: 2020-01-30

Study Results

Results available

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Basic Information

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Recruitment Status

COMPLETED

Total Enrollment

219 participants

Study Classification

OBSERVATIONAL

Study Start Date

2016-05-31

Study Completion Date

2018-07-31

Brief Summary

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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

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Conditions

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Leber's Hereditary Optic Neuropathy (LHON)

Study Design

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Observational Model Type

CASE_ONLY

Study Time Perspective

RETROSPECTIVE

Eligibility Criteria

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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
Minimum Eligible Age

12 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Santhera Pharmaceuticals

INDUSTRY

Sponsor Role lead

Responsible Party

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Responsibility Role SPONSOR

Principal Investigators

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Thomas Klopstock, Prof. Dr.

Role: PRINCIPAL_INVESTIGATOR

Friedrich-Baur-Institut, Muenchen, Germany

Locations

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CHU Saint-Pierre

Brussels, , Belgium

Site Status

Cliniques Universitaire Saint-Luc

Brussels, , Belgium

Site Status

Universitair Ziekenhuis Gent

Ghent, , Belgium

Site Status

C. H. U. Sart Tilman

Liège, , Belgium

Site Status

CHU Angers - Hôpital Hôtel Dieu

Angers, , France

Site Status

Hopital Roger Salengro - CHU Lille

Lille, , France

Site Status

Fondation Ophtalmologique Adolphe de Rothschild

Paris, , France

Site Status

CHU Reims - Hôpital Robert Debré

Reims, , France

Site Status

CHU Strasbourg - Hôpital Hautepierre

Strasbourg, , France

Site Status

Justus-Liebig-Universitaet Giessen

Giessen, , Germany

Site Status

Universitaetsklinikum Heidelberg

Heidelberg, , Germany

Site Status

Friedrich-Baur-Institut

München, , Germany

Site Status

Azienda Ospedaliera Universitaria Policlinico G. Martino

Messina, , Italy

Site Status

Fondazione IRCCS Istituto Neurologico Carlo Besta

Milan, , Italy

Site Status

Università di Pisa

Pisa, , Italy

Site Status

G. B. Bietti Fondazione - IRCCS

Rome, , Italy

Site Status

Maastricht University Medical Center

Maastricht, , Netherlands

Site Status

Oogziekenhuis Rotterdam

Rotterdam, , Netherlands

Site Status

Haukeland Universitetssykehus

Bergen, , Norway

Site Status

University Hospital of Wales

Cardiff, , United Kingdom

Site Status

Queen's Hospital

London, , United Kingdom

Site Status

Countries

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Belgium France Germany Italy Netherlands Norway United Kingdom

Provided Documents

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Document Type: Study Protocol

View Document

Document Type: Statistical Analysis Plan

View Document

Other Identifiers

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SNT-CRS-002

Identifier Type: -

Identifier Source: org_study_id

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