Post Authorisation Safety Study With Raxone in LHON Patients

NCT02771379 | Completed

• 1 other identifier: SNT-IV-003
• Study Type: observational
• Target: 229
• Locations: 6 countries
• Sites: 26

Brief Summary

This study is a multicentre, prospective, non-interventional post-authorisation safety study (PASS) of the clinical outcomes for patients with LHON treated with Raxone®. No medication will be provided as part of this study. Raxone® will be obtained through commercial channels.

Conditions

Leber's Hereditary Optic Neuropathy (LHON)

Outcome Measures

Primary Outcomes (1)

• Long-term safety profile of Raxone® in the treatment of patients with LHON when used under conditions of routine clinical care.

  Safety will be assessed by the collection and analysis of adverse events of special interest (AESIs), frequency and nature of AEs and serious adverse events (SAEs), adverse drug reactions (ADRs) and serious adverse drug reactions (SADRs)

  up to 5 years

Secondary Outcomes (1)

• Long-term effectiveness of Raxone® in the treatment of patients with LHON when used under conditions of routine clinical care

  up to 5 years

Study Arms (1)

Patients who are treated with Raxone®

Drug: Idebenone

Interventions

Idebenone DRUG

Raxone 900mg/day as per Raxone SmPC and medical judgement of treating physician.

Also known as: Raxone®

Patients who are treated with Raxone®

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

Patient prescribed Raxone® for the treatment of LHON who have completed the informed consent. This study will be conducted in EU in approximately 45 highly specialized centres in ophthalmology and neuroophthalmology.

You may qualify if:

• Patient prescribed Raxone® for the treatment of LHON;
• Patient has completed an Informed Consent Form (ICF) indicating that he/she (or a legally acceptable representative) has been informed of all pertinent aspects of the study and has agreed to participate in the study;
• Patient is not participating in any interventional study.

Sponsors & Collaborators

• Santhera Pharmaceuticals: lead

Study Sites (26)

Universitaetsklinikum Graz

Graz, 8036, Austria

Location

Medical University of Vienna

Vienna, Austria

Location

CHU de Nîmes - Hôpital Carémeau

Nîmes, Gard, 30029, France

Location

CHU Angers - Hôpital Hôtel Dieu

Angers, Maine Et Loire, 49033, France

Location

Hopital Roger Salengro - CHU Lille

Lille, Nord, 59037, France

Location

Hôpital Européen Georges Pompidou

Paris, Paris Cedex 15, 75908, France

Location

CHU Amiens - Centre Saint Victor

Amiens, Somme, 80054, France

Location

Hospices Civils de Lyon

Lyon, 69002, France

Location

Hopital Neurologique Pierre Wertheimer

Lyon, 69677, France

Location

Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts

Paris, 75012, France

Location

Fondation Ophtalmologique Adolphe de Rothschild

Paris, 75019, France

Location

Julius Maximilians University

Würzburg, Bavaria, 97070, Germany

Location

Universitaetsklinikum Essen

Essen, Germany

Location

Universitaetsklinikum Freiburg

Freiburg im Breisgau, Germany

Location

Universitaetsklinikum Hamburg-Eppendorf

Hamburg, 20246, Germany

Location

Universitaetsklinikum Heidelberg

Heidelberg, Germany

Location

Friedrich-Baur-Institut

Munich, Germany

Location

Universitaetsklinikum Muenster

Münster, Germany

Location

Dietrich-Bonhoeffer-Klinikum Neubrandenburg

Neubrandenburg, Germany

Location

Universitaetsklinikum Regensburg

Regensburg, Germany

Location

Athens Ophthalmological Center

Athens, 11528, Greece

Location

Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche

Bologna, 40123, Italy

Location

Ospedale San Raffaele

Milan, 20132, Italy

Location

Azienda Ospedaliera S. Camillo Forlanini

Rome, 00152, Italy

Location

Universitair Medisch Centrum Groningen

Groningen, 9713 GZ, Netherlands

Location

Maastricht University Medical Center

Maastricht, 6211 LK, Netherlands

Location

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber

Interventions

idebenone

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 Design

• Study Type: observational
• Observational Model: COHORT
• Time Perspective: PROSPECTIVE
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 25, 2016
• First Posted: May 13, 2016
• Study Start: September 1, 2016
• Primary Completion: April 16, 2021
• Study Completion: April 16, 2021
• Last Updated: July 16, 2021

Record last verified: 2021-07

Locations

FR (9); GR (1); NL (2); IT (3); AU (2); DE (9)