Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy (CICLO-NOHL)

• ClinicalTrials.gov Identifier: NCT02176733
• Recruitment Status: Unknown
• First Posted: June 27, 2014
• Last Update Posted: June 27, 2014
• Sponsor: University Hospital, Angers
• Information provided by (Responsible Party): University Hospital, Angers

Tracking Information

• First Submitted Date: June 25, 2014
• First Posted Date: June 27, 2014
• Last Update Posted Date: June 27, 2014
• Study Start Date: July 2011
• Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)
• Current Primary Outcome Measures (submitted: June 26, 2014): Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales [ Time Frame: at 9 months ]
• Original Primary Outcome Measures: Same as current
• Change History: No Changes Posted
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy
• Official Title: Not Provided

Brief 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. 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.

• Detailed Description: Not Provided
• Study Type: Interventional
• Study Phase: Phase 2
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment
• Condition: Leber Hereditary Optic Neuropathy
• Intervention: Drug: cyclosporine

Study Arms

Experimental: cyclosporine

Intervention: Drug: cyclosporine

• 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.

Recruitment Information

• Recruitment Status: Unknown status
• Estimated Enrollment (submitted: June 26, 2014): 12
• Original Estimated Enrollment: Same as current
• Study Completion Date: Not Provided
• Estimated Primary Completion Date: October 2015 (Final data collection date for primary outcome measure)

Eligibility Criteria

Inclusion Criteria:

• patient with the mutation confirmed by molecular analysis
• patient with a recent loss of monocular vision (≤ 6 months)
• voluntarily Patient Consent

Exclusion Criteria:

• 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: France

Administrative Information

• NCT Number: NCT02176733
• Other Study ID Numbers: PHRC 2010-05
• Has Data Monitoring Committee: Not Provided
• U.S. FDA-regulated Product: Not Provided
• IPD Sharing Statement: Not Provided
• Current Responsible Party: University Hospital, Angers
• Original Responsible Party: Same as current
• Current Study Sponsor: University Hospital, Angers
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: University Hospital, Angers
• Verification Date: January 2014