Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey

NCT01892943 | Completed

• 1 other identifier: LHON HCR
• Study Type: observational
• Target: 306
• Locations: 5 countries
• Sites: 8

Brief Summary

The objective of this survey is to establish the clinical course of vision loss and recovery in patients with a genetically confirmed diagnosis of Leber Hereditary Optic Neuropathy (LHON). Visual acuity changes over time from onset of symptoms and from visual acuity nadir will be the main endpoint analysed. The survey will collect historically documented visual acuity data for all patients at participating sites with a genetically confirmed diagnosis of LHON. No exclusion criteria apply. Patients are not required to attend the clinic for the survey. Data will be collected in a completely anonymous manner. Ethical approvals and data release agreements will be obtained as required by local regulations.

Conditions

Leber Hereditary Optic Neuropathy (LHON)

Outcome Measures

Primary Outcomes (1)

• Visual acuity

  The Entire duration of the disease patient by patient basis up to the date of completion of the Case Report Form (CRF).

  average 0 to 10 years (retrospective)

Study Arms (1)

Patients with LHON

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)
• Sampling Method: Non-Probability Sample

Study Population

patients with genetically confirmed diagnosis of LHON

You may qualify if:

• patients with genetically confirmed diagnosis of LHON

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead
• European Vision Institute Clinical Research Network: collaborator

Study Sites (8)

University Hospitals Leuven

Leuven, 3000, Belgium

Location

Glostrup Hospital/National Eye Clinic of the Kennedy Center/University of Copenhagen Department of Ophthalmology

Glostrup Municipality, 2600, Denmark

Location

CHU Bordeaux

Bordeaux, 33000, France

Location

CIC/CMR CHNO des Quinze-Vingts

Paris, 75012, France

Location

Universty of Bari

Bari, 70100, Italy

Location

Fondazione G.B. Bietti

Rome, 000199, Italy

Location

San Raffaele Hospital

Segrate, 20132, Italy

Location

University Eye Clinic

Ljubljana, 1000, Slovenia

Location

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber

Condition Hierarchy (Ancestors)

Optic Atrophies, Hereditary; Optic Atrophy; Optic Nerve Diseases; Cranial Nerve Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Eye Diseases, Hereditary; Eye Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Officials

• Jose-Alain Sahel, MD

  Centre de Recherche Institut de la Vision INSERMN

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: observational
• Observational Model: CASE ONLY
• Time Perspective: RETROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 26, 2013
• First Posted: July 8, 2013
• Study Start: August 1, 2013
• Primary Completion: February 1, 2014
• Study Completion: February 1, 2014
• Last Updated: May 15, 2014

Record last verified: 2014-05

Locations

BE (1); SL (1); FR (2); DK (1); IT (3)