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

Leber's Hereditary Optic Neuropathy (LHON) Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02796274
• Other study ID #: SNT-CRS-002
• Status: Completed
• Start date: May 2016
• Est. completion date: July 2018

Study information

• Verified date: January 2020
• Source: Santhera Pharmaceuticals
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 219
• Est. completion date: July 2018
• Est. primary completion date: March 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 12 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Leber's Hereditary Optic Neuropathy (LHON)
• Optic Atrophy, Hereditary, Leber
• Optic Nerve Diseases

Locations

Country	Name	City	State
Belgium	Cliniques Universitaire Saint-Luc	Brussel
Belgium	CHU Saint-Pierre	Brussels
Belgium	Universitair Ziekenhuis Gent	Gent
Belgium	C. H. U. Sart Tilman	Liege
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
Germany	Justus-Liebig-Universitaet Giessen	Giessen
Germany	Universitaetsklinikum Heidelberg	Heidelberg
Germany	Friedrich-Baur-Institut	Muenchen
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
Netherlands	Maastricht University Medical Center	Maastricht
Netherlands	Oogziekenhuis Rotterdam	Rotterdam
Norway	Haukeland Universitetssykehus	Bergen
United Kingdom	University Hospital of Wales	Cardiff
United Kingdom	Queen's Hospital	London

Sponsors (1)

Lead Sponsor	Collaborator
Santhera Pharmaceuticals

Countries where clinical trial is conducted

Belgium,  France,  Germany,  Italy,  Netherlands,  Norway,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	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