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The purpose of this survey is to collect visual acuity data from patients with LHON in order to establish the clinical course (natural history) and visual acuity outcomes in patients with a genetically confirmed diagnosis of LHON. In addition, this survey will generate data that will serve as comparator for the open-label study SNT-IV-006.
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| Measure | Description | Time Frame |
|---|---|---|
| In Eyes With VA Assessment Made ≤1 Year After Onset of Symptoms: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA (Measured by Change in ETDRS Letters) From Baseline (BL) or in Which BL VA Better Than 1.0 logMAR Was Maintained at 12 Months | Clinically Relevant Recovery (CRR) is defined as a VA improvement from off-chart (ETDRS) to 5 letters on-chart, or an on-chart improvement of 10 letters. | 12 months |
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Inclusion Criteria:
Exclusion Criteria:
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Patients with a genetically confirmed diagnosis of LHON
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| Name | Affiliation | Role |
|---|---|---|
| Thomas Klopstock, Prof. Dr. | Friedrich-Baur-Institut, Muenchen, Germany | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| CHU Saint-Pierre | Brussels | Belgium | ||||
| Cliniques Universitaire Saint-Luc |
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| ID | Title | Description |
|---|---|---|
| FG000 | LHON Patients Case Record Cohort | Historical case record data from all LHON patients fulfilling the prospectively-defined inclusion criteria:
|
| Title | Milestones | Reasons Not Completed | |||||
|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 5, 2016 | Oct 1, 2019 |
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| Brussels |
| Belgium |
| Universitair Ziekenhuis Gent | Ghent | Belgium |
| C. H. U. Sart Tilman | Liège | Belgium |
| CHU Angers - Hôpital Hôtel Dieu | Angers | France |
| Hopital Roger Salengro - CHU Lille | Lille | France |
| Fondation Ophtalmologique Adolphe de Rothschild | Paris | France |
| CHU Reims - Hôpital Robert Debré | Reims | France |
| CHU Strasbourg - Hôpital Hautepierre | Strasbourg | France |
| Justus-Liebig-Universitaet Giessen | Giessen | Germany |
| Universitaetsklinikum Heidelberg | Heidelberg | Germany |
| Friedrich-Baur-Institut | München | Germany |
| Azienda Ospedaliera Universitaria Policlinico G. Martino | Messina | Italy |
| Fondazione IRCCS Istituto Neurologico Carlo Besta | Milan | Italy |
| Università di Pisa | Pisa | Italy |
| G. B. Bietti Fondazione - IRCCS | Rome | Italy |
| Maastricht University Medical Center | Maastricht | Netherlands |
| Oogziekenhuis Rotterdam | Rotterdam | Netherlands |
| Haukeland Universitetssykehus | Bergen | Norway |
| University Hospital of Wales | Cardiff | United Kingdom |
| Queen's Hospital | London | United Kingdom |
| COMPLETED |
|
| NOT COMPLETED |
|
Visual Acuity (VA) at Baseline (logMAR)
| ID | Title | Description |
|---|---|---|
| BG000 | LHON Patients Case Record Cohort | Historical case record data from all LHON patients fulfilling the prospectively-defined inclusion criteria:
Actual: 48 (with VA assessment made ≤1 year after the onset of symptoms) out of 219 patients from whom data were collected and entered into the database. |
| Units | Counts |
|---|---|
| Participants |
|
| eyes |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes | ||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants | Participants | Participants |
|
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| Age, Continuous | Median | Inter-Quartile Range | years | Participants |
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| Sex: Female, Male | Count of Participants | Participants | Participants |
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| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants | Participants |
| ||||||||||||||||||||||||
| Region of Enrollment | Number | participants | Participants |
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| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | In Eyes With VA Assessment Made ≤1 Year After Onset of Symptoms: Proportion of Eyes With Clinically Relevant Recovery (CRR) of VA (Measured by Change in ETDRS Letters) From Baseline (BL) or in Which BL VA Better Than 1.0 logMAR Was Maintained at 12 Months | Clinically Relevant Recovery (CRR) is defined as a VA improvement from off-chart (ETDRS) to 5 letters on-chart, or an on-chart improvement of 10 letters. | Data from a total of 219 LHON patients and 438 eyes were obtained from this study, out of which 96 eyes had a VA assessment made ≤1 year after the onset of symptoms (primary outcome measure). | Posted | Median | Inter-Quartile Range | ETDRS letters | 12 months | eyes | eyes |
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No safety data was collected
No safety data was collected
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | LHON Patients Case Record Cohort | Historical case record data from all LHON patients fulfilling the prospectively-defined inclusion criteria:
Actual: 48 (with VA assessment made ≤1 year after the onset of symptoms) out of 219 patients from whom data were collected and entered into the database. | 0 | 0 | 0 | 0 | 0 | 0 |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Vanessa Dos Reis Ferreira | Santhera Pharmaceuticals | +41 61 906 89 50 | office@santhera.com |
| Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Apr 5, 2016 | Oct 1, 2019 | SAP_001.pdf |
| ID | Term |
|---|---|
| D029242 | Optic Atrophy, Hereditary, Leber |
| ID | Term |
|---|---|
| D015418 | Optic Atrophies, Hereditary |
| D009896 | Optic Atrophy |
| D009901 | Optic Nerve Diseases |
| D003389 | Cranial Nerve Diseases |
| D009422 | Nervous System Diseases |
| D020271 | Heredodegenerative Disorders, Nervous System |
| D019636 | Neurodegenerative Diseases |
| D015785 | Eye Diseases, Hereditary |
| D005128 | Eye Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D028361 | Mitochondrial Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| Title | Measurements |
|---|---|
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| Title | Measurements |
|---|---|
| Female |
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| Male |
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| Title | Measurements |
|---|---|
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| Belgium |
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| Poland |
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| Italy |
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| United Kingdom |
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| France |
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| Germany |
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