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

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ClinicalTrials.gov Identifier: NCT02796274
Recruitment Status : Completed
First Posted : June 10, 2016
Last Update Posted : October 4, 2018
Sponsor:
Information provided by (Responsible Party):
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.

Condition or disease
Leber's Hereditary Optic Neuropathy (LHON)

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Study Type : Observational
Actual Enrollment : 219 participants
Observational Model: Case-Only
Time Perspective: Retrospective
Official Title: Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
Study Start Date : May 2016
Actual Primary Completion Date : March 2018
Actual Study Completion Date : July 2018





Primary Outcome Measures :
  1. Proportion of eyes with clinically relevant recovery or clinically relevant stabilization of VA [ Time Frame: 12 months ]
    In eyes with a VA assessment made ≤1 year after the onset of symptoms: Proportion of eyes with clinically relevant recovery of VA from Baseline or in which Baseline VA better than 1.0 logarithm of the minimal angle of resolution (logMAR) was maintained at 12 months (primary time point)



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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with a genetically confirmed diagnosis of LHON
Criteria

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

Information from the National Library of Medicine

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): NCT02796274


Locations
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Belgium
CHU Saint-Pierre
Brussels, Belgium
Cliniques Universitaire Saint-Luc
Brussel, Belgium
Universitair Ziekenhuis Gent
Gent, Belgium
C. H. U. Sart Tilman
Liege, Belgium
France
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
Germany
Justus-Liebig-Universitaet Giessen
Giessen, Germany
Universitaetsklinikum Heidelberg
Heidelberg, Germany
Friedrich-Baur-Institut
Muenchen, Germany
Italy
Azienda Ospedaliera Universitaria Policlinico G. Martino
Messina, Italy
Fondazione IRCCS Istituto Neurologico Carlo Besta
Milano, Italy
Università di Pisa
Pisa, Italy
G. B. Bietti Fondazione - IRCCS
Rome, Italy
Netherlands
Maastricht University Medical Center
Masstricht, Netherlands
Oogziekenhuis Rotterdam
Rotterdam, Netherlands
Norway
Haukeland Universitetssykehus
Bergen, Norway
United Kingdom
University Hospital of Wales
Cardiff, United Kingdom
Queen's Hospital
Essex, United Kingdom
Sponsors and Collaborators
Santhera Pharmaceuticals
Investigators
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Principal Investigator: Thomas Klopstock, Prof. Dr. Friedrich-Baur-Institut, Muenchen, Germany

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Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02796274     History of Changes
Other Study ID Numbers: SNT-CRS-002
First Posted: June 10, 2016    Key Record Dates
Last Update Posted: October 4, 2018
Last Verified: October 2018

Additional relevant MeSH terms:
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Optic Nerve Diseases
Optic Neuritis
Optic Atrophy, Hereditary, Leber
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases