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

• Sponsor: University Hospital, Angers (Other)
• Overall Status: Unknown status
• CT.gov ID: NCT02176733
• Collaborator: (none)
• Enrollment: 12
• Location: 1
• Arm: 1

Study Details

Study Description

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.

Condition or Disease	Intervention/Treatment	Phase
Leber Hereditary Optic Neuropathy	Drug: cyclosporine	Phase 2

Study Design

• Study Type: Interventional
• Anticipated Enrollment: 12 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Study Start Date: Jul 1, 2011
• Anticipated Primary Completion Date: Oct 1, 2015

Arms and Interventions

Arm	Intervention/Treatment
Experimental: cyclosporine	Drug: cyclosporine

Outcome Measures

Primary Outcome Measures

1. Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales [at 9 months]

Eligibility Criteria

Criteria

• Ages Eligible for Study: 18 Years and Older
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Centre Hospitalier Universitaire	Angers		France	49000

Sponsors and Collaborators

• University Hospital, Angers

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

• Responsible Party: University Hospital, Angers
• ClinicalTrials.gov Identifier: NCT02176733
• Other Study ID Numbers: PHRC 2010-05
• First Posted: Jun 27, 2014
• Last Update Posted: Jun 27, 2014
• Last Verified: Jan 1, 2014

• Additional relevant MeSH terms: Optic Nerve Diseases; Optic Atrophy, Hereditary, Leber; Cyclosporine; Cyclosporins

Study Results

No Results Posted as of Jun 27, 2014