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

NCT ID: NCT02176733

Last Updated: 2014-06-27

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 12 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2011-07-31

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.

Conditions

Leber Hereditary Optic Neuropathy

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

cyclosporine

Group Type: EXPERIMENTAL

cyclosporine

Intervention Type: DRUG

Interventions

cyclosporine

Intervention Type: DRUG

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

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

University Hospital, Angers

OTHER_GOV

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Locations

Centre Hospitalier Universitaire

Angers, , France

Site Status: RECRUITING

Countries

France

Central Contacts

D Milea

Role: CONTACT

damilea@chu-angers.fr

Facility Contacts

D Milea

Role: primary

damilea@chu-angers.fr

References

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.

Reference Type: DERIVED

PMID: 29454364 (View on PubMed)

Other Identifiers

PHRC 2010-05

Identifier Type: -

Identifier Source: org_study_id