Leber Hereditary Optic NeuropathyPhase 2CICLO-NOHLNCT02176733
Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy
A Phase 2 study testing cyclosporine in people with Leber Hereditary Optic Neuropathy, run by University Hospital, Angers at 1 site in France.
Trial summary
The Leber Hereditary Optic Neuropathy is a genetic disorder caused by maternal transmission of mitochondrial DesoxiroboNucleid Acid mutations. It is manifested by a rapidly progressive blindness, profound, due to atrophic optic nerve. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Leber Hereditary Optic Neuropathy
- What is being tested?
- cyclosporine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 12 participants
- Where
- 1 siteFrance
- Study dates
- Jul 2011to Oct 2015
- Phase
- Phase 2
- 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 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- patient with the mutation confirmed by molecular analysis
- patient with a recent loss of monocular vision (≤ 6 months)
- voluntarily Patient Consent
Who cannot join
- patient who have not given their written and informed consent signed
- against indication of cyclosporine
- no drug compliance to previous inclusion
- no national health insurance affiliation
- pregnant women or lactating
- women who could become pregnant during the study period and with no contraception
- private patients of their liberty by judicial or administrative decision, or patients under supervision
Trial locations and contacts
1 site in 1 country.
D Milea contact
France1 site, 1 recruiting
- Recruiting
Centre Hospitalier Universitaire
Angers, 49000
D Milea, damilea@chu-angers.fr
About this study
The Leber Hereditary Optic Neuropathy is a genetic disorder caused by maternal transmission of mitochondrial DesoxiroboNucleid Acid mutations. It is manifested by a rapidly progressive blindness, profound, due to atrophic optic nerve. The visual loss is primarily unilateral bilateralisation taking place in the vast majority of cases in weeks or months. The neuro-cardio-protective properties of cyclosporine (and its analogs specifically targeting the anti-apoptotic mechanisms) are particularly promising.
The investigators hypothesis is that cyclosporine may limit apoptosis during the acute phase of the disease process and would limit the loss of visual acuity and improve the visual prognosis of these patients.
- Conditions
- Leber Hereditary Optic Neuropathy
- Being tested
- cyclosporine
- 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.
cyclosporineDrug
| Study group | Role | What they receive |
|---|---|---|
| cyclosporine | Experimental | Not described Receives: Drug: cyclosporine |
What is being measured
The primary outcome is the main question the study is designed to answer.
Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales
Measured: at 9 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2014 (estimated)
- Study startJul 2011
- Primary completionOct 2015 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Leber Hereditary Optic NeuropathyStudies of the same condition, open and under way.
- More trials run by University Hospital, AngersThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 12 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Angers
- Sponsor type
- Other government
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02176733
- Sponsor's protocol ID
- PHRC 2010-05
- Acronym
- CICLO-NOHL
- Status last verified
- Jan 2014
- First submitted
- 25 Jun 2014
- Last updated
- 27 Jun 2014
- Results posted
- No
Publications
Leruez S, Verny C, Bonneau D, Procaccio V, Lenaers G, Amati-Bonneau P, Reynier P, Scherer C, Prundean A, Orssaud C, Zanlonghi X, Rougier MB, Tilikete C, Milea D. Cyclosporine A does not prevent second-eye involvement in Leber's hereditary optic neuropathy. Orphanet J Rare Dis. 2018 Feb 17;13(1):33. doi: 10.1186/s13023-018-0773-y.
PubMed 29454364
Study at a glance
From the registry record, last updated by the sponsor on 27 Jun 2014. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy
- ClinicalTrials.gov ID
- NCT02176733
- Acronym / sponsor ID
- CICLO-NOHL / PHRC 2010-05
- Conditions
- Leber Hereditary Optic Neuropathy
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 12 participants (estimated)
- Lead sponsor
- University Hospital, AngersOther government
- Study start
- Jul 2011
- Primary completion
- Oct 2015 (estimated)
- First posted
- 27 Jun 2014 (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 Leber Hereditary Optic Neuropathy is a genetic disorder caused by maternal transmission of mitochondrial DesoxiroboNucleid Acid mutations. It is manifested by a rapidly progressive blindness, profound, due to atrophic optic nerve. The visual loss is primarily unilateral bilateralisation taking place in the vast majority of cases in weeks or months. The neuro-cardio-protective properties of cyclosporine (and its analogs specifically targeting the anti-apoptotic mechanisms) are particularly promising.
The investigators hypothesis is that cyclosporine may limit apoptosis during the acute phase of the disease process and would limit the loss of visual acuity and improve the visual prognosis of these patients.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| cyclosporine | Experimental | Drug: cyclosporine |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (3 listed)
- patient with the mutation confirmed by molecular analysis
- patient with a recent loss of monocular vision (≤ 6 months)
- voluntarily Patient Consent
Key exclusion criteria (7 listed)
- patient who have not given their written and informed consent signed
- against indication of cyclosporine
- no drug compliance to previous inclusion
- no national health insurance affiliation
- pregnant women or lactating
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 |
|---|---|---|
| cyclosporine | Drug | cyclosporine |
Primary outcome
Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales
Time frame: at 9 months
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 27 Jun 2014.
- Derived from this study: PubMed 29454364Leruez S, Verny C, Bonneau D, Procaccio V, Lenaers G, Amati-Bonneau P, Reynier P, Scherer C, Prundean A, Orssaud C, Zanlonghi X, Rougier MB, Tilikete C, Milea D. Cyclosporine A does not prevent second-eye involvement in Leber's hereditary optic neuropathy. Orphanet J Rare Dis. 2018 Feb 17;13(1):33. doi: 10.1186/s13023-018-0773-y.
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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.
D Milea contact
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing cyclosporine in people with Leber Hereditary Optic Neuropathy, run by University Hospital, Angers at 1 site in France.
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 7 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: status not confirmed, as of the sponsor's last update on 27 Jun 2014. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. 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.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact D Milea (damilea@chu-angers.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales (time frame: at 9 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02176733, which also lists 1 linked publication.
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 27 Jun 2014.