Leber Hereditary Optic Neuropathy (LHON)NCT01892943
Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey
An observational study of Leber Hereditary Optic Neuropathy (LHON), run by Santhera Pharmaceuticals at 8 sites in 5 countries.
Trial summary
The objective of this survey is to establish the clinical course of vision loss and recovery in patients with a genetically confirmed diagnosis of Leber Hereditary Optic Neuropathy (LHON). Visual acuity changes over time from onset of symptoms and from visual acuity nadir will be the main endpoint analysed. Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Leber Hereditary Optic Neuropathy (LHON)
- How many people
- 306 participants
- Where
- 8 sitesin 5 countries
- Study dates
- Aug 2013to Feb 2014
- 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: patients with genetically confirmed diagnosis of LHON
Who may be able to join
- patients with genetically confirmed diagnosis of LHON
Who cannot join
None listed.
Trial locations and contacts
8 sites in 5 countries.
8 sites in 5 countries
Italy3 sites
Universty of Bari
Bari, 70100
Fondazione G.B. Bietti
Rome, 000199
San Raffaele Hospital
Segrate, 20132
France2 sites
CHU Bordeaux
Bordeaux, 33000
CIC/CMR CHNO des Quinze-Vingts
Paris, 75012
Denmark1 site
Glostrup Hospital/National Eye Clinic of the Kennedy Center/University of Copenhagen Department of Ophthalmology
Glostrup Municipality, 2600
About this study
The objective of this survey is to establish the clinical course of vision loss and recovery in patients with a genetically confirmed diagnosis of Leber Hereditary Optic Neuropathy (LHON).
Visual acuity changes over time from onset of symptoms and from visual acuity nadir will be the main endpoint analysed.
Read more
The survey will collect historically documented visual acuity data for all patients at participating sites with a genetically confirmed diagnosis of LHON. No exclusion criteria apply. Patients are not required to attend the clinic for the survey.
Data will be collected in a completely anonymous manner. Ethical approvals and data release agreements will be obtained as required by local regulations.
- Conditions
- Leber Hereditary Optic Neuropathy (LHON)
- 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.
| Study group | Role | What they receive |
|---|---|---|
| Patients with LHON | Not described |
What is being measured
The primary outcome is the main question the study is designed to answer.
Visual acuity
The Entire duration of the disease patient by patient basis up to the date of completion of the Case Report Form (CRF).
Measured: average 0 to 10 years (retrospective)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted8 Jul 2013 (estimated)
- Study startAug 2013
- Primary completionFeb 2014
- Study completionFeb 2014
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Leber 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
- Case only
- Time perspective
- Retrospective
- Enrolment
- 306 participants (actual)
Sponsor and investigators
- Lead sponsor
- Santhera Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- European Vision Institute Clinical Research Network
- Responsible party
- Sponsor
- Investigators
- Jose-Alain Sahel, MD, Principal investigator, Centre de Recherche Institut de la Vision INSERMN
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01892943
- Sponsor's protocol ID
- LHON HCR
- Status last verified
- May 2014
- First submitted
- 26 Jun 2013
- Last updated
- 15 May 2014
- Results posted
- No
Study at a glance
From the registry record, last updated by the sponsor on 15 May 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Leber Hereditary Optic Neuropathy (LHON) Historical Case Record Survey
- ClinicalTrials.gov ID
- NCT01892943
- Acronym / sponsor ID
- LHON HCR
- Conditions
- Leber Hereditary Optic Neuropathy (LHON)
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 306 participants (actual)
- Lead sponsor
- Santhera PharmaceuticalsIndustry (pharma, biotech, devices)
- Collaborators
- European Vision Institute Clinical Research Network
- Study start
- Aug 2013
- Primary completion
- Feb 2014 (actual)
- Study completion
- Feb 2014 (actual)
- First posted
- 8 Jul 2013 (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.
The objective of this survey is to establish the clinical course of vision loss and recovery in patients with a genetically confirmed diagnosis of Leber Hereditary Optic Neuropathy (LHON).
Visual acuity changes over time from onset of symptoms and from visual acuity nadir will be the main endpoint analysed.
The survey will collect historically documented visual acuity data for all patients at participating sites with a genetically confirmed diagnosis of LHON. No exclusion criteria apply. Patients are not required to attend the clinic for the survey.
Data will be collected in a completely anonymous manner. Ethical approvals and data release agreements will be obtained as required by local regulations.
Study design
- Observational model
- Case only
- Time perspective
- Retrospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Patients with LHON | – | – |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- patients with genetically confirmed diagnosis of LHON
Key inclusion criteria (1 listed)
- patients with genetically confirmed diagnosis of LHON
Key exclusion criteria (0 listed)
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Visual acuity
Time frame: average 0 to 10 years (retrospective)
The Entire duration of the disease patient by patient basis up to the date of completion of the Case Report Form (CRF).
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 15 May 2014.
Related trials for Leber Hereditary Optic Neuropathy (LHON)
Not yet recruitingPhase 1/2
A Basket Clinical Study to Assess Glycerol Tributyrate in Patients With Mitochondrial Encephalopathy, Lactic Acidosis, Stroke-like Episodes (MELAS) or Leber's Hereditary Optic Neuropathy-Plus (LHON-Plus)
MELAS Syndrome, Lebers Hereditory Optic Neuropathy With Extra Ocular Symptoms (LHON-Plus)
Recruiting
New Non-invasive Modalities for Assessing Retinal Structure and Function
Ischemic Optic Neuropathy, Branch Retinal Artery Occlusion
Recruiting
Stem Cell Ophthalmology Treatment Study II
Retinal Disease, Age-Related Macular Degeneration
RecruitingPhase 2
Efficacy and Safety Study of Bilateral IVT Injection of GS010 at Two Dose Levels in LHON Patients
Leber Hereditary Optic Disease
Active, not recruitingPhase 3
A Phase 3, Multicenter, Randomized, Double-Masked, Sham-Controlled Clinical Trial for Leber's Hereditary Optic Neuropathy (LHON) Associated With ND4 Mutation
Leber Hereditary Optic Neuropathy (LHON)
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.
Jose-Alain Sahel, MD principal investigator
Centre de Recherche Institut de la Vision INSERMN
8 sites in 5 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Leber Hereditary Optic Neuropathy (LHON), run by Santhera Pharmaceuticals at 8 sites in 5 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 1 inclusion and 0 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 15 May 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 8 sites in Italy, France, Belgium, Denmark, Slovenia. 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?
Visual acuity (time frame: average 0 to 10 years (retrospective)). 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 15 May 2014.