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

Hôpital Necker-Enfants Malades14 enrolled

Overview

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

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

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Safety Issues Efficacy, Self

Interventions

BéfizalDRUG

600 mf befizal a day for one year

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

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

Locations (1)

HEGP Hospital

Paris, France

Outcomes

Primary Outcomes

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

Time frame: Month12

Secondary Outcomes

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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Data from ClinicalTrials.gov

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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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: 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

Time frame: Month 15

Evolution of retinal nerve fibers layer Optical Coherent Tomography

physiological parameter : Optical Coherent Tomography

Time frame: Month 6

Evolution of retinal nerve fibers layer Optical Coherent Tomography

physiological parameter : Optical Coherent Tomography

Time frame: 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)

Time frame: Month 3

automated visual field measurement

physiological parameter : automated visual field

Time frame: 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)

Time frame: 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)

Time frame: 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)

Time frame: Month 15

Manual visual field measurement

physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

Time frame: Month 3

Manual visual field measurement

physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

Time frame: Month 6

Manual visual field measurement

physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

Time frame: Month 9

Manual visual field measurement

physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

Time frame: Month 12

Manual visual field measurement

physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

Time frame: Month 15

National Eye Institute Visual Function Questionnaire 25

Questionnaire

Time frame: Month 12

Concentration of serum creatinine