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

January 20, 2020 updated by: Santhera Pharmaceuticals
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.

Study Overview

Status

Completed

Study Type

Observational

Enrollment (Actual)

219

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

      • Brussel, Belgium
        • Cliniques Universitaire Saint-Luc
      • Brussels, Belgium
        • CHU Saint-Pierre
      • Gent, Belgium
        • Universitair Ziekenhuis Gent
      • Liege, Belgium
        • C. H. U. Sart Tilman
      • Angers, France
        • CHU Angers - Hôpital Hôtel Dieu
      • Lille, France
        • Hopital Roger Salengro - CHU Lille
      • Paris, France
        • Fondation Ophtalmologique Adolphe de Rothschild
      • Reims, France
        • CHU Reims - Hôpital Robert Debré
      • Strasbourg, France
        • CHU Strasbourg - Hôpital Hautepierre
      • Giessen, Germany
        • Justus-Liebig-Universitaet Giessen
      • Heidelberg, Germany
        • Universitaetsklinikum Heidelberg
      • Muenchen, Germany
        • Friedrich-Baur-Institut
      • Messina, Italy
        • Azienda Ospedaliera Universitaria Policlinico G. Martino
      • Milano, Italy
        • Fondazione IRCCS Istituto Neurologico Carlo Besta
      • Pisa, Italy
        • Università di Pisa
      • Rome, Italy
        • G. B. Bietti Fondazione - IRCCS
      • Maastricht, Netherlands
        • Maastricht University Medical Center
      • Rotterdam, Netherlands
        • Oogziekenhuis Rotterdam
      • Bergen, Norway
        • Haukeland Universitetssykehus
      • Cardiff, United Kingdom
        • University Hospital of Wales
      • London, United Kingdom
        • Queen's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

10 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

Sampling Method

Non-Probability Sample

Study Population

Patients with a genetically confirmed diagnosis of LHON

Description

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 Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Case-Only
  • Time Perspectives: Retrospective

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
Time Frame: 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.
12 months

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Investigators

  • Principal Investigator: Thomas Klopstock, Prof. Dr., Friedrich-Baur-Institut, Muenchen, Germany

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

May 1, 2016

Primary Completion (Actual)

March 1, 2018

Study Completion (Actual)

July 1, 2018

Study Registration Dates

First Submitted

June 7, 2016

First Submitted That Met QC Criteria

June 7, 2016

First Posted (Estimate)

June 10, 2016

Study Record Updates

Last Update Posted (Actual)

January 30, 2020

Last Update Submitted That Met QC Criteria

January 20, 2020

Last Verified

January 1, 2020

More Information

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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