Leber's Hereditary Optic Neuropathy (LHON)PAROSNCT02771379
Post Authorisation Safety Study With Raxone in LHON Patients
An observational study of Leber's Hereditary Optic Neuropathy (LHON), run by Santhera Pharmaceuticals at 26 sites in 6 countries.
Trial 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. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Leber's Hereditary Optic Neuropathy (LHON)
- What is being tested?
- Idebenone
- How many people
- 229 participants
- Where
- 26 sitesin 6 countries
- Study dates
- Sep 2016to 16 Apr 2021
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- 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.
Who cannot join
- No explicit exclusion criteria exist to avoid selection bias and to allow for documentation of routine clinical practice.
Trial locations and contacts
26 sites in 6 countries.
26 sites in 6 countries
France9 sites
CHU Amiens - Centre Saint Victor
Amiens, Somme, 80054
CHU Angers - Hôpital Hôtel Dieu
Angers, Maine Et Loire, 49033
Hopital Roger Salengro - CHU Lille
Lille, Nord, 59037
Hospices Civils de Lyon
Lyon, 69002
Hopital Neurologique Pierre Wertheimer
Lyon, 69677
CHU de Nîmes - Hôpital Carémeau
Nîmes, Gard, 30029
Hôpital Européen Georges Pompidou
Paris, Paris Cedex 15, 75908
Centre Hospitalier National d'Ophtalmologie (CHNO) des Quinze-Vingts
Paris, 75012
Fondation Ophtalmologique Adolphe de Rothschild
Paris, 75019
Germany9 sites
Universitaetsklinikum Essen
Essen
Universitaetsklinikum Freiburg
Freiburg im Breisgau
Universitaetsklinikum Hamburg-Eppendorf
Hamburg, 20246
Universitaetsklinikum Heidelberg
Heidelberg
Friedrich-Baur-Institut
Munich
Universitaetsklinikum Muenster
Münster
Dietrich-Bonhoeffer-Klinikum Neubrandenburg
Neubrandenburg
Universitaetsklinikum Regensburg
Regensburg
Julius Maximilians University
Würzburg, Bavaria, 97070
Italy3 sites
Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche
Bologna, 40123
Ospedale San Raffaele
Milan, 20132
Azienda Ospedaliera S. Camillo Forlanini
Rome, 00152
Austria2 sites
Universitaetsklinikum Graz
Graz, 8036
Medical University of Vienna
Vienna
Netherlands2 sites
Universitair Medisch Centrum Groningen
Groningen, 9713 GZ
Maastricht University Medical Center
Maastricht, 6211 LK
About this study
Official title: 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.
- Being tested
- Idebenone
- Medical subject headings
- Optic Atrophy, Hereditary, Leber
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
IdebenoneDrug
Also called Raxone®
Raxone 900mg/day as per Raxone SmPC and medical judgement of treating physician.
| Study group | Role | What they receive |
|---|---|---|
| Patients who are treated with Raxone® | Not described Receives: Drug: Idebenone |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 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
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.
Measured: up to 5 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 May 2016 (estimated)
- Study startSep 2016
- Primary completion16 Apr 2021
- Study completion16 Apr 2021
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leber's Hereditary Optic Neuropathy (LHON)Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Cohort
- Time perspective
- Prospective
- Enrolment
- 229 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02771379
- Sponsor's protocol ID
- SNT-IV-003
- Acronym
- PAROS
- Status last verified
- Jul 2021
- First submitted
- 25 Apr 2016
- Last updated
- 16 Jul 2021
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 16 Jul 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Non-interventional Study of Clinical Experience in Patients Prescribed Raxone® for the Treatment of Leber's Hereditary Optic Neuropathy (LHON)
- ClinicalTrials.gov ID
- NCT02771379
- Acronym / sponsor ID
- PAROS / SNT-IV-003
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 229 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Sep 2016
- Primary completion
- 16 Apr 2021 (actual)
- Study completion
- 16 Apr 2021 (actual)
- First posted
- 13 May 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients who are treated with Raxone® | – | Drug: Idebenone |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (3 listed)
- 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.
Key exclusion criteria (1 listed)
- No explicit exclusion criteria exist to avoid selection bias and to allow for documentation of routine clinical practice.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IdebenoneAlso: Raxone®Raxone 900mg/day as per Raxone SmPC and medical judgement of treating physician. | Drug | Patients who are treated with Raxone® |
Primary outcome
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)
1 secondary outcome
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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 16 Jul 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
26 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leber's Hereditary Optic Neuropathy (LHON), run by Santhera Pharmaceuticals at 26 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 3 inclusion and 1 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 16 Jul 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 26 sites in France, Germany, Italy, Austria, Netherlands and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 16 Jul 2021.