Clinical trial NCT02796274: Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
Official title: Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
Study type: OBSERVATIONAL
Overall status: COMPLETED
Conditions: Leber's Hereditary Optic Neuropathy (LHON)
Lead sponsor: Santhera Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2016-06-10
Enrollment: 219
Sex: ALL
Minimum age (years): 12
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: RETROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Patients with a genetically confirmed diagnosis of LHON

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.


Eligibility 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

Primary outcomes:
measure: 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
timeFrame: 12 months
Secondary outcomes:
Other outcomes:
Locations:
city: Brussels
country: Belgium
facility: CHU Saint-Pierre
city: Brussel
country: Belgium
facility: Cliniques Universitaire Saint-Luc
city: Gent
country: Belgium
facility: Universitair Ziekenhuis Gent
city: Liege
country: Belgium
facility: C. H. U. Sart Tilman
city: Angers
country: France
facility: CHU Angers - Hôpital Hôtel Dieu
city: Lille
country: France
facility: Hopital Roger Salengro - CHU Lille
city: Paris
country: France
facility: Fondation Ophtalmologique Adolphe de Rothschild
city: Reims
country: France
facility: CHU Reims - Hôpital Robert Debré
city: Strasbourg
country: France
facility: CHU Strasbourg - Hôpital Hautepierre
city: Giessen
country: Germany
facility: Justus-Liebig-Universitaet Giessen
city: Heidelberg
country: Germany
facility: Universitaetsklinikum Heidelberg
city: Muenchen
country: Germany
facility: Friedrich-Baur-Institut
city: Messina
country: Italy
facility: Azienda Ospedaliera Universitaria Policlinico G. Martino
city: Milano
country: Italy
facility: Fondazione IRCCS Istituto Neurologico Carlo Besta
city: Pisa
country: Italy
facility: Università di Pisa
city: Rome
country: Italy
facility: G. B. Bietti Fondazione - IRCCS
city: Maastricht
country: Netherlands
facility: Maastricht University Medical Center
city: Rotterdam
country: Netherlands
facility: Oogziekenhuis Rotterdam
city: Bergen
country: Norway
facility: Haukeland Universitetssykehus
city: Cardiff
country: United Kingdom
facility: University Hospital of Wales
city: London
country: United Kingdom
facility: Queen's Hospital
Study officials:
affiliation: Friedrich-Baur-Institut, Muenchen, Germany
name: Thomas Klopstock, Prof. Dr.
role: PRINCIPAL_INVESTIGATOR
References: