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

Status

Indicates the current recruitment status or the expanded access status.
Completed

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Observational

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
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
        • Hopital 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 Europeén 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

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

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

Group / Cohort

A group or subgroup of participants in an observational study that is assessed for biomedical or health outcomes.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
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

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
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

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
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

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
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

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
September 1, 2016

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
April 16, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
April 16, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
April 25, 2016

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
May 10, 2016

First Posted (Estimate)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
May 13, 2016

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
July 16, 2021

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
July 15, 2021

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2021

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant 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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