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

NCT02176733 | Unknown Phase 2

• 1 other identifier: PHRC 2010-05
• Study Type: interventional
• Target: 12
• Locations: 1 country
• Sites: 1

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

Outcome Measures

Primary Outcomes (1)

• Measurement of visual acuity with Monoyer, Early Treatment Diabetic Retinopathy Study and Parinaud scales

  at 9 months

Study Arms (1)

cyclosporine

EXPERIMENTAL

Drug: cyclosporine

Interventions

cyclosporine DRUG

cyclosporine

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

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

You may not qualify if:

• patient who have not given their written and informed consent signed
• against indication of cyclosporine
• 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

Sponsors & Collaborators

• University Hospital, Angers: lead

Study Sites (1)

Centre Hospitalier Universitaire

Angers, 49000, France

RECRUITING

Related Publications (1)

• 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.

  PMID: 29454364 DERIVED

MeSH Terms

Conditions

Optic Atrophy, Hereditary, Leber

Interventions

Cyclosporine

Condition Hierarchy (Ancestors)

Optic Atrophies, Hereditary; Optic Atrophy; Optic Nerve Diseases; Cranial Nerve Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Eye Diseases, Hereditary; Eye Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Mitochondrial Diseases; Metabolic Diseases; Nutritional and Metabolic Diseases

Intervention Hierarchy (Ancestors)

Cyclosporins; Peptides, Cyclic; Macrocyclic Compounds; Polycyclic Compounds; Peptides; Amino Acids, Peptides, and Proteins

Central Study Contacts

D Milea

CONTACT

damilea@chu-angers.fr

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER GOV
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2014
• First Posted: June 27, 2014
• Study Start: July 1, 2011
• Primary Completion: October 1, 2015
• Last Updated: June 27, 2014

Record last verified: 2014-01

Locations

FR (1)