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

July 15, 2021 updated by: Santhera Pharmaceuticals

A Non-interventional Study of Clinical Experience in Patients Prescribed Raxone® for the Treatment of Leber's Hereditary Optic Neuropathy (LHON)

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Observational

Enrollment (Actual)

229

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Austria
      • Graz, Austria, 8036
        • Universitaetsklinikum Graz
      • Vienna, Austria
        • Medical University of Vienna
  • France
      • Lyon, France, 69002
        • Hospices Civils de Lyon
      • Lyon Cedex, France, 69677
        • Hôpital Neurologique Pierre Wertheimer
      • Paris, France, 75019
        • Fondation Ophtalmologique Adolphe de Rothschild
      • Paris, France, 75012
        • Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts
    • Gard
      • Nîmes, Gard, France, 30029
        • CHU de Nîmes - Hôpital Caremeau
    • Maine Et Loire
      • Angers, Maine Et Loire, France, 49033
        • CHU Angers - Hôpital Hôtel Dieu
    • Nord
      • Lille, Nord, France, 59037
        • Hopital Roger Salengro - CHU Lille
    • Paris Cedex 15
      • Paris, Paris Cedex 15, France, 75908
        • Hôpital Européen Georges Pompidou
    • Somme
      • Amiens, Somme, France, 80054
        • CHU Amiens - Centre Saint Victor
  • Germany
      • Essen, Germany
        • Universitaetsklinikum Essen
      • Freiburg, Germany
        • Universitaetsklinikum Freiburg
      • Hamburg, Germany, 20246
        • Universitaetsklinikum Hamburg-Eppendorf
      • Heidelberg, Germany
        • Universitaetsklinikum Heidelberg
      • Munich, Germany
        • Friedrich-Baur-Institut
      • Munster, Germany
        • Universitaetsklinikum Muenster
      • Neubrandenburg, Germany
        • Dietrich-Bonhoeffer-Klinikum Neubrandenburg
      • Regensburg, Germany
        • Universitaetsklinikum Regensburg
    • Bavaria
      • Wuerzburg, Bavaria, Germany, 97070
        • Julius Maximilians University
  • Greece
      • Athens, Greece, 11528
        • Athens Ophthalmological Center
  • Italy
      • Bologna, Italy, 40123
        • Universita di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
      • Milano, Italy, 20132
        • Ospedale San Raffaele
      • Rome, Italy, 00152
        • Azienda Ospedaliera S. Camillo Forlanini
  • Netherlands
      • Groningen, Netherlands, 9713 GZ
        • Universitair Medisch Centrum Groningen
      • Maastricht, Netherlands, 6211 LK
        • Maastricht University Medical Center

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Child
  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Genders Eligible for Study

All

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.

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Observational Models: Cohort
  • Time Perspectives: Prospective

Cohorts and Interventions

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 Names:
  • Raxone®

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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)
up to 5 years

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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.
up to 5 years

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Santhera Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

September 1, 2016

Primary Completion (Actual)

April 16, 2021

Study Completion (Actual)

April 16, 2021

Study Registration Dates

First Submitted

April 25, 2016

First Submitted That Met QC Criteria

May 10, 2016

First Posted (Estimate)

May 13, 2016

Study Record Updates

Last Update Posted (Actual)

July 16, 2021

Last Update Submitted That Met QC Criteria

July 15, 2021

Last Verified

July 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • SNT-IV-003

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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