Post Authorisation Safety Study With Raxone in LHON Patients (PAROS)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. Identifier: NCT02771379
Recruitment Status : Recruiting
First Posted : May 13, 2016
Last Update Posted : October 4, 2018
Information provided by (Responsible Party):
Santhera Pharmaceuticals

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.

Condition or disease Intervention/treatment
Leber's Hereditary Optic Neuropathy (LHON) Drug: Idebenone

Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: A Non-interventional Study of Clinical Experience in Patients Prescribed Raxone® for the Treatment of Leber's Hereditary Optic Neuropathy (LHON)
Study Start Date : September 2016
Estimated Primary Completion Date : February 2021
Estimated Study Completion Date : February 2021

Group/Cohort Intervention/treatment
Patients who are treated with Raxone® Drug: Idebenone
Raxone 900mg/day as per Raxone SmPC and medical judgement of treating physician.
Other Name: Raxone®

Primary Outcome Measures :
  1. Long-term safety profile of Raxone® in the treatment of patients with LHON when used under conditions of routine clinical care. [ Time Frame: up to 5 years ]
    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)

Secondary Outcome Measures :
  1. Long-term effectiveness of Raxone® in the treatment of patients with LHON when used under conditions of routine clinical care [ Time Frame: up to 5 years ]
    According to the Summary of Product Characteristics (SmPC) a patient's vision should be continually monitored therefore effectiveness will be assessed by measuring on-therapy changes in patients' visual acuity (VA), visual fields (VF), colour contrast sensitivity and Retinal Nerve Fibre Layer thickness (RNFL) where available.

Information from the National Library of Medicine

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Ages Eligible for Study:   Child, Adult, Older Adult
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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.

Inclusion Criteria:

  • 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.

Exclusion Criteria:

  • No explicit exclusion criteria exist to avoid selection bias and to allow for documentation of routine clinical practice.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT02771379

Contact: Vanessa Ferreira

Universitaetsklinikum Graz Recruiting
Graz, Austria, 8036
Principal Investigator: Mona Schneider, MD         
Medical University of Vienna Recruiting
Vienna, Austria
Principal Investigator: Berthold Pemp, MD         
CHU de Nîmes - Hôpital Carémeau Recruiting
Nîmes, Gard, France, 30029
Principal Investigator: Luc Jeanjean, MD         
CHU Angers - Hôpital Hôtel Dieu Recruiting
Angers, Maine Et Loire, France, 49033
Principal Investigator: Christophe Verny, MD         
Hopital Roger Salengro - CHU Lille Recruiting
Lille, Nord, France, 59037
Principal Investigator: Sabine Defoort Delemmes, MD         
Hôpital Européen Georges Pompidou Recruiting
Paris, Paris Cedex 15, France, 75908
Principal Investigator: Christophe Orssaud, MD         
CHU Amiens - Centre Saint Victor Recruiting
Amiens, Somme, France, 80054
Principal Investigator: Solange Milazzo, MD         
Hopital Neurologique Pierre Wertheimer Recruiting
Lyon Cedex, France, 69677
Principal Investigator: Damien Biotti, MD         
Hospices Civils de Lyon Recruiting
Lyon, France, 69002
Principal Investigator: Caroline Tilikete, MD         
Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts Recruiting
Paris, France, 75012
Principal Investigator: Catherine Vignal, MD         
Fondation Ophtalmologique Adolphe de Rothschild Recruiting
Paris, France, 75019
Principal Investigator: Rabih Hage, MD         
Julius Maximilians University Recruiting
Wuerzburg, Baden Wuerttemberg, Germany, 97070
Principal Investigator: Thomas Ach, MD         
Charite - Campus Virchow-Klinikum Recruiting
Berlin, Germany, 13353
Principal Investigator: Uwe Pleyer, MD         
Universitaetsklinikum Essen Recruiting
Essen, Germany
Principal Investigator: Michael Oeverhaus, MD         
Universitaetsklinikum Freiburg Recruiting
Freiburg, Germany
Principal Investigator: Wolf Lagreze         
Universitaetsklinikum Hamburg-Eppendorf Recruiting
Hamburg, Germany, 20246
Principal Investigator: Martin Spitzer, MD         
Universitaetsklinikum Heidelberg Recruiting
Heidelberg, Germany
Principal Investigator: Klaus Rohrschneider, MD         
Friedrich-Baur-Institut Recruiting
Munich, Germany
Principal Investigator: Thomas Klopstock         
Universitaetsklinikum Muenster Recruiting
Munster, Germany
Principal Investigator: Nicole Eter, MD         
Universitaetsklinikum Neubrandenburg Recruiting
Neubrandenburg, Germany
Principal Investigator: Herbert Jaegle, MD         
Universitaetsklinikum Regensburg Recruiting
Regensburg, Germany
Principal Investigator: Helmut Hoeh, MD         
Athens Ophthalmological Center Recruiting
Athens, Greece, 11528
Principal Investigator: Agathi Kouri, MD         
Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche Not yet recruiting
Bologna, Italy, 40123
Principal Investigator: Valerio Carelli, MD         
Ospedale San Raffaele Recruiting
Milano, Italy, 20132
Principal Investigator: Francesco Maria Bandello, MD         
Azienda Ospedaliera S. Camillo Forlanini Not yet recruiting
Rome, Italy, 00152
Principal Investigator: Anna De Negri, MD         
Universitair Medisch Centrum Groningen Recruiting
Groningen, Netherlands, 9713 GZ
Principal Investigator: Jan-Willem Pott, MD         
Maastricht University Medical Center Not yet recruiting
Maastricht, Netherlands, 6211 LK
Principal Investigator: Noël Bauer, MD         
Sponsors and Collaborators
Santhera Pharmaceuticals

Responsible Party: Santhera Pharmaceuticals Identifier: NCT02771379     History of Changes
Other Study ID Numbers: SNT-IV-003
First Posted: May 13, 2016    Key Record Dates
Last Update Posted: October 4, 2018
Last Verified: October 2018

Additional relevant MeSH terms:
Optic Nerve Diseases
Optic Atrophy, Hereditary, Leber
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Molecular Mechanisms of Pharmacological Action
Protective Agents
Physiological Effects of Drugs