## Protocol Section
### Identification Module
**NCT ID:** NCT02771379

**Acronym:** PAROS

**Brief Title:** Post Authorisation Safety Study With Raxone in LHON Patients

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

#### Organization Study ID Info
**ID:** SNT-IV-003
#### Organization
**Class:** INDUSTRY
**Full Name:** Santhera Pharmaceuticals
### Status Module
#### Completion Date
**Date:** 2021-04-16

**Type:** ACTUAL

#### Expanded Access Info
#### Last Update Post Date
**Date:** 2021-07-16

**Type:** ACTUAL

**Last Update Submit Date:** 2021-07-15

**Overall Status:** COMPLETED

#### Primary Completion Date
**Date:** 2021-04-16

**Type:** ACTUAL

#### Start Date
**Date:** 2016-09

**Status Verified Date:** 2021-07

#### Study First Post Date
**Date:** 2016-05-13
**Type:** ESTIMATED
**Study First Submit Date:** 2016-04-25
**Study First Submit QC Date:** 2016-05-10
### Sponsor Collaborators Module

#### Lead Sponsor

**Class:** INDUSTRY

**Name:** Santhera Pharmaceuticals

#### Responsible Party

**Type:** SPONSOR

### Description Module

**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 Module

**Conditions:**

- Leber's Hereditary Optic Neuropathy (LHON)

### Design Module

#### Design Info

**Observational Model:** COHORT

**Time Perspective:** PROSPECTIVE

#### Enrollment Info

**Count:** 229

**Type:** ACTUAL

**Study Type:** OBSERVATIONAL

### Arms Interventions Module

#### Arm Group 1

**Intervention Names:**

- Drug: Idebenone

**Label:** Patients who are treated with Raxone®

### Interventions

#### Intervention 1

**Arm Group Labels:**

- Patients who are treated with Raxone®

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

**Name:** Idebenone

**Other Names:**

- Raxone®

**Type:** DRUG

### Outcomes Module

#### Primary Outcomes

**Description:** 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)

**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

#### Secondary Outcomes

**Description:** 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.

**Measure:** 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

### Eligibility Module

**Eligibility Criteria:** 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.

**Sampling Method:** NON_PROBABILITY_SAMPLE

**Sex:** ALL

**Standard Ages:**

- 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.

### Contacts Locations Module

#### Locations

**Location 1:**

**City:** Graz

**Country:** Austria

**Facility:** Universitaetsklinikum Graz

**Zip:** 8036

**Location 2:**

**City:** Vienna

**Country:** Austria

**Facility:** Medical University of Vienna

**Location 3:**

**City:** Nîmes

**Country:** France

**Facility:** CHU de Nîmes - Hôpital Carémeau

**State:** Gard

**Zip:** 30029

**Location 4:**

**City:** Angers

**Country:** France

**Facility:** CHU Angers - Hôpital Hôtel Dieu

**State:** Maine Et Loire

**Zip:** 49033

**Location 5:**

**City:** Lille

**Country:** France

**Facility:** Hopital Roger Salengro - CHU Lille

**State:** Nord

**Zip:** 59037

**Location 6:**

**City:** Paris

**Country:** France

**Facility:** Hôpital Européen Georges Pompidou

**State:** Paris Cedex 15

**Zip:** 75908

**Location 7:**

**City:** Amiens

**Country:** France

**Facility:** CHU Amiens - Centre Saint Victor

**State:** Somme

**Zip:** 80054

**Location 8:**

**City:** Lyon Cedex

**Country:** France

**Facility:** Hopital Neurologique Pierre Wertheimer

**Zip:** 69677

**Location 9:**

**City:** Lyon

**Country:** France

**Facility:** Hospices Civils de Lyon

**Zip:** 69002

**Location 10:**

**City:** Paris

**Country:** France

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

**Zip:** 75012

**Location 11:**

**City:** Paris

**Country:** France

**Facility:** Fondation Ophtalmologique Adolphe de Rothschild

**Zip:** 75019

**Location 12:**

**City:** Wuerzburg

**Country:** Germany

**Facility:** Julius Maximilians University

**State:** Bavaria

**Zip:** 97070

**Location 13:**

**City:** Essen

**Country:** Germany

**Facility:** Universitaetsklinikum Essen

**Location 14:**

**City:** Freiburg

**Country:** Germany

**Facility:** Universitaetsklinikum Freiburg

**Location 15:**

**City:** Hamburg

**Country:** Germany

**Facility:** Universitaetsklinikum Hamburg-Eppendorf

**Zip:** 20246

**Location 16:**

**City:** Heidelberg

**Country:** Germany

**Facility:** Universitaetsklinikum Heidelberg

**Location 17:**

**City:** Munich

**Country:** Germany

**Facility:** Friedrich-Baur-Institut

**Location 18:**

**City:** Munster

**Country:** Germany

**Facility:** Universitaetsklinikum Muenster

**Location 19:**

**City:** Neubrandenburg

**Country:** Germany

**Facility:** Dietrich-Bonhoeffer-Klinikum Neubrandenburg

**Location 20:**

**City:** Regensburg

**Country:** Germany

**Facility:** Universitaetsklinikum Regensburg

**Location 21:**

**City:** Athens

**Country:** Greece

**Facility:** Athens Ophthalmological Center

**Zip:** 11528

**Location 22:**

**City:** Bologna

**Country:** Italy

**Facility:** Università di Bologna-Clinica Neurologica-Dipartimento di Scienze Neurologiche

**Zip:** 40123

**Location 23:**

**City:** Milano

**Country:** Italy

**Facility:** Ospedale San Raffaele

**Zip:** 20132

**Location 24:**

**City:** Rome

**Country:** Italy

**Facility:** Azienda Ospedaliera S. Camillo Forlanini

**Zip:** 00152

**Location 25:**

**City:** Groningen

**Country:** Netherlands

**Facility:** Universitair Medisch Centrum Groningen

**Zip:** 9713 GZ

**Location 26:**

**City:** Maastricht

**Country:** Netherlands

**Facility:** Maastricht University Medical Center

**Zip:** 6211 LK

## Derived Section

### Condition Browse Module - Ancestors

- ID: D000003389

- Term: Cranial Nerve Diseases

- ID: D000009422

- Term: Nervous System Diseases

- ID: D000005128

- Term: Eye Diseases

- ID: D000015418

- Term: Optic Atrophies, Hereditary

- ID: D000009896

- Term: Optic Atrophy

- ID: D000020271

- Term: Heredodegenerative Disorders, Nervous System

- ID: D000019636

- Term: Neurodegenerative Diseases

- ID: D000015785

- Term: Eye Diseases, Hereditary

- ID: D000030342

- Term: Genetic Diseases, Inborn

- ID: D000028361

- Term: Mitochondrial Diseases

- ID: D000008659

- Term: Metabolic Diseases

### Condition Browse Module - Browse Branches

- Abbrev: BC10

- Name: Nervous System Diseases

- Abbrev: BC11

- Name: Eye Diseases

- Abbrev: All

- Name: All Conditions

- Abbrev: BC16

- Name: Diseases and Abnormalities at or Before Birth

- Abbrev: BC18

- Name: Nutritional and Metabolic Diseases

- Abbrev: BC23

- Name: Symptoms and General Pathology

- Abbrev: Rare

- Name: Rare Diseases

### Condition Browse Module - Browse Leaves

- ID: M12832

- Name: Optic Nerve Diseases

- Relevance: HIGH

- As Found: Optic Neuropathy

- ID: M23422

- Name: Optic Atrophy, Hereditary, Leber

- Relevance: HIGH

- As Found: Leber's Hereditary Optic Neuropathy

- ID: M4589

- Name: Atrophy

- Relevance: LOW

- As Found: Unknown

- ID: M12827

- Name: Optic Atrophy

- Relevance: LOW

- As Found: Unknown

- ID: M18093

- Name: Optic Atrophies, Hereditary

- Relevance: LOW

- As Found: Unknown

- ID: M6605

- Name: Cranial Nerve Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M8271

- Name: Eye Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M22092

- Name: Heredodegenerative Disorders, Nervous System

- Relevance: LOW

- As Found: Unknown

- ID: M21558

- Name: Neurodegenerative Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M18339

- Name: Eye Diseases, Hereditary

- Relevance: LOW

- As Found: Unknown

- ID: M23686

- Name: Genetic Diseases, Inborn

- Relevance: LOW

- As Found: Unknown

- ID: M23341

- Name: Mitochondrial Diseases

- Relevance: LOW

- As Found: Unknown

- ID: M11639

- Name: Metabolic Diseases

- Relevance: LOW

- As Found: Unknown

- ID: T3352

- Name: Leber Hereditary Optic Neuropathy

- Relevance: HIGH

- As Found: Leber's Hereditary Optic Neuropathy

### Condition Browse Module - Meshes

- ID: D000009901

- Term: Optic Nerve Diseases

- ID: D000029242

- Term: Optic Atrophy, Hereditary, Leber

### Intervention Browse Module - Ancestors

- ID: D000000975

- Term: Antioxidants

- ID: D000045504

- Term: Molecular Mechanisms of Pharmacological Action

- ID: D000020011

- Term: Protective Agents

- ID: D000045505

- Term: Physiological Effects of Drugs

### Intervention Browse Module - Browse Branches

- Abbrev: All

- Name: All Drugs and Chemicals

### Intervention Browse Module - Browse Leaves

- ID: M273909

- Name: Idebenone

- Relevance: HIGH

- As Found: Pulmonary Nodules

- ID: M4292

- Name: Antioxidants

- Relevance: LOW

- As Found: Unknown

- ID: M21869

- Name: Protective Agents

- Relevance: LOW

- As Found: Unknown

### Intervention Browse Module - Meshes

- ID: C000036619

- Term: Idebenone

### Misc Info Module

- Version Holder: 2024-05-24
