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

Study Overview
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Brief Summary
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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

Detailed Description
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Study of the efficiency of Béfizal® 200 mg in 14 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

Official Title
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Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy

Conditions
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Safety Issues, Efficacy, Self

Intervention / Treatment
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* Drug: Béfizal


Participation Criteria
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Eligibility Criteria
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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

Ages Eligible for Study
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Minimum Age: 18 Years

Sexes Eligible for Study
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All

Accepts Healthy Volunteers
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No

Study Plan
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How is the study designed?
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Design Details

Primary Purpose: Treatment
Intervention Model: Single Group Assignment
Interventional Model Description: open
Masking: None (Open Label)


Arms and Interventions

| Participant Group/Arm | Intervention/Treatment |
| --- | --- |
| Experimental: treatment group<br>14 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. Befizal® 200 mg will be tested for one year | Drug: Béfizal<br>* 600 mf befizal a day for one year<br>|

What is the study measuring?
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Primary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Evolution of best corrected farsight visual acuity (in LogMAR) | Measurement of the best corrected farsight visual acuity by Early Treatment Diabetic Retinopathy Study type scale (range from -0.3 to 2.6) -0.3 is the best vision and 2.6 the worse | Month12 | 

Secondary Outcome Measures

| Outcome Measure | Measure Description | Time Frame |
| --- | --- | --- |
| Farsight best corrected visual acuity | Measurement of the Farsight best corrected visual acuity in LogMAR by Early Treatment Diabetic Retinopathy Study type scale (range from -0.3 to 2.6) -0.3 is the best vision and 2.6 the worse | Month 3 | 
| Evolution of Farsight best corrected visual acuity | Measurement of the Farsight best corrected visual acuity in LogMAR by Early Treatment Diabetic Retinopathy Study type scale (range from -0.3 to 2.6) -0.3 is the best vision and 2.6 the worse | Month 6 | 
| Evolution of Farsight best corrected visual acuity | Measurement of the Farsight best corrected visual acuity in LogMAR by Early Treatment Diabetic Retinopathy Study type scale (range from -0.3 to 2.6) -0.3 is the best vision and 2.6 the worse | Month 9 | 
| Evolution of Farsight best corrected visual acuity | Measurement of the Farsight best corrected visual acuity in LogMAR by Early Treatment Diabetic Retinopathy Study type scale (range from -0.3 to 2.6) -0.3 is the best vision and 2.6 the worse | Month 15 | 
| Evolution of Farsight best corrected visual acuity | Measurement of the Farsight best corrected visual acuity by Early Treatment Diabetic Retinopathy Study type scale (range from -0.3 to 2.6) -0.3 is the best vision and 2.6 the worse | Month 3 | 
| Evolution of Farsight decimal best corrected visual acuity | Measurement of the Farsight best corrected visual acuity (in LogMAR) measured with a Monoyer scale (range from 20/20 to light perception). 20/20 is the best vision and light perception the worse | Month 6 | 
| Evolution of Farsight decimal best corrected visual acuity | Measurement of the Farsight best corrected visual acuity (in LogMAR) measured with a Monoyer scale (range from 20/20 to light perception). 20/20 is the best vision and light perception the worse | Month 9 | 
| Evolution of Farsight decimal best corrected visual acuity | Measurement of the Farsight best corrected visual acuity (in LogMAR) measured with a Monoyer scale (range from 20/20 to light perception). 20/20 is the best vision and light perception the worse | Month 12 | 
| Evolution of Farsight decimal best corrected visual acuity | Measurement of the Farsight best corrected visual acuity (in LogMAR) measured with a Monoyer scale (range from 20/20 to light perception). 20/20 is the best vision and light perception the worse | Month 15 | 
| Evolution of Nearsight visual acuity | Measurement of the best corrected nearsight visual acuity measured using a Parinaud scale (from P48 to P1>.5) P48 is the worse vision and P1.5 the best | Month 3 | 
| Evolution of Nearsight visual acuity | Measurement of the best corrected nearsight visual acuity measured using a Parinaud scale (from P48 to P1>.5) P48 is the worse vision and P1.5 the best | Month 6 | 
| Evolution of Nearsight visual acuity | Measurement of the best corrected nearsight visual acuity measured using a Parinaud scale (from P48 to P1>.5)P48 is the worse vision and P1.5 the best | Month 9 | 
| Evolution of Nearsight visual acuity | Measurement of the best corrected nearsight visual acuity measured using a Parinaud scale (from P48 to P1>.5) P48 is the worse vision and P1.5 the best | Month 12 | 
| Evolution of Nearsight visual acuity | Measurement of the best corrected nearsight visual acuity measured using a Parinaud scale (from P48 to P1>.5) P48 is the worse vision and P1.5 the best | Month 15 | 
| Evolution of retinal nerve fibers layer Optical Coherent Tomography | physiological parameter : Optical Coherent Tomography | Month 6 | 
| Evolution of retinal nerve fibers layer Optical Coherent Tomography | physiological parameter : Optical Coherent Tomography | Month 12 | 
| automated visual field measurement | physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France) | Month 3 | 
| automated visual field measurement | physiological parameter : automated visual field | Month 6 | 
| automated visual field | physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France) | Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0 | 
| automated visual field measurement | physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France) | Month 12 | 
| automated visual field measurement | physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France) | Month 15 | 
| Manual visual field measurement | physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1 | Month 3 | 
| Manual visual field measurement | physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1 | Month 6 | 
| Manual visual field measurement | physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1 | Month 9 | 
| Manual visual field measurement | physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1 | Month 12 | 
| Manual visual field measurement | physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1 | Month 15 | 
| National Eye Institute Visual Function Questionnaire 25 | Questionnaire | Month 12 | 
| Concentration of serum creatinine | Concentration of serum creatinine | Every three months until Month 15 | 
| Concentration of high-density lipoprotein cholesterol | Blood test | Every three months until Month 15 | 
| Concentration of very low-density lipoprotein cholesterol | Blood test | Every three months until Month 15 | 
| Concentration of triglycerides | Blood test | Every three months until Month 15 | 
| Rate of Partial thromboplastin time | Blood test | Every three months until Month 15 | 
| Concentration of Aspartate aminotransférase | Blood test | Every three months until Month 15 | 
| Concentration of Alanine aminotransférase | Blood test | Every three months until Month 15 | 
| Concentration of lactate deshydrogenase | Blood test | Every three months until Month 15 | 
| Concentration of creatine kinase | Blood test | Every three months until Month 15 | 

 Terms related to the study
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Keywords Provided by Centre Hospitalier Valida
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leber herediatry optic neuropathy, befizal