Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy

• Status: Recruiting
• Trial end date: 2023-03-10
• Target enrollment: 0
• Participant gender: All

Summary

Study of the efficiency of Béfizal® 200 mg in 14 adult patients with a LHON that occurred for less than 5 years. Patient must have certain specific mutations

• Phase: Phase 2/Phase 3
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Hôpital Necker-Enfants Malades
• Collaborators: CLAIROP; European Georges Pompidou Hospital
• Treatments: Bezafibrate

Criteria

Inclusion Criteria:

• Adult patients in whom the diagnosis of LHON obtained on anamnestic, clinical and ancillary testing / laboratory data. LHON should have occurred for less than 5 years and must be genetically proved with a 3460 or 11778 mitochondrial DNA mutation. Given the mode of transmission, genetic research may have been carried out in a maternal relative

Exclusion Criteria:

• * Any optic neuropathy for which the diagnosis of LHON is not formally confirmed or genetically proven;

• LHON that started for more than 5 years;

• LHON associated with another primary mutation than 3460 or 11778

• Children or adult patients under guardianship or deprived of liberty by administrative or judicial decision;

• Women of childbearing age ; pregnant or lactating women;

• Patients who do not have affiliation to a social protection scheme (national or private insurance / beneficiary or assignee);

• Patient who did not give its written, informed and signed consent;

• Allergy to fibrate, bezafibrate and / or BEFIZAL® 200mg (Arrow Generiques) or one of these constituents;

• Photosensitivity reactions related to fibrates;

• Patient already receiving treatment with fibrates or HMG Co-A reductase inhibitors or anticoagulants;

• Hepatic insuffisiency or dysfunction with increased of transaminases (AST and ALT) over 3 times of the normal;

• Renal insufficiency with serum creatinine> 15 mg / L (> 135 mg / dL) Biliary pathology