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Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)

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ClinicalTrials.gov Identifier: NCT02796274
Recruitment Status : Completed
First Posted : June 10, 2016
Results First Posted : January 30, 2020
Last Update Posted : January 30, 2020
Sponsor:
Information provided by (Responsible Party):
Santhera Pharmaceuticals

Brief Summary:
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.

Condition or disease
Leber's Hereditary Optic Neuropathy (LHON)

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Study Type : Observational
Actual Enrollment : 219 participants
Observational Model: Case-Only
Time Perspective: Retrospective
Official Title: Historical Case Record Survey of Visual Acuity Data From Patients With Leber's Hereditary Optic Neuropathy (LHON)
Study Start Date : May 2016
Actual Primary Completion Date : March 2018
Actual Study Completion Date : July 2018





Primary Outcome Measures :
  1. 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 [ Time Frame: 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.



Information from the National Library of Medicine

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Ages Eligible for Study:   12 Years and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Non-Probability Sample
Study Population
Patients with a genetically confirmed diagnosis of LHON
Criteria

Inclusion Criteria:

  1. age ≥ 12 years
  2. the onset of symptoms is dated after 1999 and is well documented (at least month of onset of symptoms is known for each eye)
  3. at least two VA assessments are available within 5 years of onset of symptoms and prior to idebenone use
  4. have a genetic diagnosis for LHON for one of the following mitochondrial DNA (mtDNA) mutations: G11778A, G3460A, T14484C

Exclusion Criteria:

  1. any participation in an interventional clinical trial after the onset of symptoms
  2. any other cause of visual impairment (e.g. glaucoma, diabetic retinopathy, AIDS related visual impairment, cataract, macular degeneration, etc.) or any active ocular disorder (uveitis, infections, inflammatory retinal disease, thyroid eye disease, etc.) during the data collection period

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02796274


Locations
Show Show 21 study locations
Sponsors and Collaborators
Santhera Pharmaceuticals
Investigators
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Principal Investigator: Thomas Klopstock, Prof. Dr. Friedrich-Baur-Institut, Muenchen, Germany
  Study Documents (Full-Text)

Documents provided by Santhera Pharmaceuticals:
Study Protocol  [PDF] April 5, 2016
Statistical Analysis Plan  [PDF] April 5, 2016

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Responsible Party: Santhera Pharmaceuticals
ClinicalTrials.gov Identifier: NCT02796274    
Other Study ID Numbers: SNT-CRS-002
First Posted: June 10, 2016    Key Record Dates
Results First Posted: January 30, 2020
Last Update Posted: January 30, 2020
Last Verified: January 2020
Additional relevant MeSH terms:
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Optic Nerve Diseases
Optic Atrophy, Hereditary, Leber
Nervous System Diseases
Cranial Nerve Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases