Trial of Cyclosporine in the Acute Phase of Leber Hereditary Optic Neuropathy

Leber Hereditary Optic Neuropathy Clinical Trial

— CICLO-NOHL  

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02176733
• Other study ID #: PHRC 2010-05
• Status: Recruiting
• Phase: Phase 2
• First received: June 25, 2014
• Last updated: June 26, 2014
• Start date: July 2011

Study information

• Verified date: January 2014
• Source: University Hospital, Angers
• Contact: D Milea
• Email: damilea[@]chu-angers.fr
• Is FDA regulated: No
• Health authority: France: Ministry of Health
• Study type: Interventional

Clinical 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. 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 12
• Est. primary completion date: October 2015
• Accepts healthy volunteers: No
• Gender: Both
• Age group: 18 Years and older

Eligibility

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

Study Design

Intervention Model: Single Group Assignment, Masking: Open Label, Primary Purpose: Treatment

Related Conditions & MeSH terms

• Leber Hereditary Optic Neuropathy
• Optic Atrophy, Hereditary, Leber
• Optic Nerve Diseases

Intervention

Drug:
• cyclosporine

Locations

Country	Name	City	State
France	Centre Hospitalier Universitaire	Angers

Sponsors (1)

Lead Sponsor	Collaborator
University Hospital, Angers

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales		at 9 months	Yes