- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02796274
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
Conditions
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
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Brussel, Belgium
- Cliniques Universitaire Saint-Luc
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Brussels, Belgium
- CHU Saint-Pierre
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Gent, Belgium
- Universitair Ziekenhuis Gent
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Liege, Belgium
- C. H. U. Sart Tilman
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Angers, France
- CHU Angers - Hôpital Hôtel Dieu
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Lille, France
- Hopital Roger Salengro - CHU Lille
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Paris, France
- Fondation Ophtalmologique Adolphe de Rothschild
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Reims, France
- CHU Reims - Hôpital Robert Debré
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Strasbourg, France
- CHU Strasbourg - Hôpital Hautepierre
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Giessen, Germany
- Justus-Liebig-Universitaet Giessen
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Heidelberg, Germany
- Universitaetsklinikum Heidelberg
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Muenchen, Germany
- Friedrich-Baur-Institut
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Messina, Italy
- Azienda Ospedaliera Universitaria Policlinico G. Martino
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Milano, Italy
- Fondazione IRCCS Istituto Neurologico Carlo Besta
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Pisa, Italy
- Università di Pisa
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Rome, Italy
- G. B. Bietti Fondazione - IRCCS
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Maastricht, Netherlands
- Maastricht University Medical Center
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Rotterdam, Netherlands
- Oogziekenhuis Rotterdam
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Bergen, Norway
- Haukeland Universitetssykehus
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Cardiff, United Kingdom
- University Hospital of Wales
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London, United Kingdom
- Queen's Hospital
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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:
- age ≥ 12 years
- the onset of symptoms is dated after 1999 and is well documented (at least month of onset of symptoms is known for each eye)
- at least two VA assessments are available within 5 years of onset of symptoms and prior to idebenone use
- have a genetic diagnosis for LHON for one of the following mitochondrial DNA (mtDNA) mutations: G11778A, G3460A, T14484C
Exclusion Criteria:
- any participation in an interventional clinical trial after the onset of symptoms
- 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 |
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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
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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.
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12 months
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
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
Terms related to this study
Additional Relevant MeSH Terms
- Metabolic Diseases
- Nervous System Diseases
- Eye Diseases
- Genetic Diseases, Inborn
- Neurodegenerative Diseases
- Eye Diseases, Hereditary
- Heredodegenerative Disorders, Nervous System
- Cranial Nerve Diseases
- Optic Atrophies, Hereditary
- Optic Atrophy
- Mitochondrial Diseases
- Optic Nerve Diseases
- Optic Atrophy, Hereditary, Leber
Other Study ID Numbers
- SNT-CRS-002
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.
Clinical Trials on Leber's Hereditary Optic Neuropathy (LHON)
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Santhera PharmaceuticalsCompletedLeber's Hereditary Optic Neuropathy (LHON)Italy, Germany, France, Austria, Greece, Netherlands
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Wuhan Neurophth Biotechnology Limited CompanyRecruitingLeber's Hereditary Optic Neuropathy (LHON)China
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Medical College of WisconsinTerminatedLeber's Hereditary Optic Neuropathy (LHON)United States
-
Santhera PharmaceuticalsCompletedLeber's Hereditary Optic Neuropathy (LHON)United States, United Kingdom, Spain, Poland, Austria, Belgium, Bulgaria, Germany, Italy, Portugal
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Argo SanteGenSight BiologicsCompleted
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Santhera PharmaceuticalsEuropean Vision Institute Clinical Research NetworkCompletedLeber Hereditary Optic Neuropathy (LHON)Belgium, France, Denmark, Italy, Slovenia
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Neurophth Therapeutics IncRecruitingLeber Hereditary Optic Neuropathy (LHON)China, United States
-
Neurophth Therapeutics IncRecruitingLeber Hereditary Optic Neuropathy (LHON)United States
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University Hospital, AngersUnknownLeber Hereditary Optic NeuropathyFrance
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GenSight BiologicsCompletedLeber Hereditary Optic NeuropathyUnited States, France, Italy, United Kingdom, Germany