Efficacy, Self, Safety Issues
Conditions
Keywords
leber herediatry optic neuropathy, befizal
Brief 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
Detailed description
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
Interventions
DRUGBéfizal
600 mf befizal a day for one year
Sponsors
European Georges Pompidou Hospital
CLAIROP
Hôpital Necker-Enfants Malades
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Intervention model description
open
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
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
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Evolution of best corrected farsight visual acuity (in LogMAR) | Month12 | 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 |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Farsight best corrected visual acuity | Month 3 | 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 |
| Evolution of Farsight best corrected visual acuity | Month 6 | 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 |
| Evolution of Farsight decimal best corrected visual acuity | Month 6 | 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 |
| Manual visual field measurement | Month 3 | physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1 |
| Evolution of Nearsight visual acuity | Month 3 | 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 |
| Evolution of retinal nerve fibers layer Optical Coherent Tomography | Month 6 | physiological parameter : Optical Coherent Tomography |
| automated visual field measurement | Month 3 | physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France) |
| automated visual field | Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0 | physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France) |
| National Eye Institute Visual Function Questionnaire 25 | Month 12 | Questionnaire |
| Concentration of high-density lipoprotein cholesterol | Every three months until Month 15 | Blood test |
| Concentration of very low-density lipoprotein cholesterol | Every three months until Month 15 | Blood test |
| Concentration of triglycerides | Every three months until Month 15 | Blood test |
| Rate of Partial thromboplastin time | Every three months until Month 15 | Blood test |
| Concentration of Aspartate aminotransférase | Every three months until Month 15 | Blood test |
| Concentration of Alanine aminotransférase | Every three months until Month 15 | Blood test |
| Concentration of lactate deshydrogenase | Every three months until Month 15 | Blood test |
| Concentration of creatine kinase | Every three months until Month 15 | Blood test |
| Concentration of serum creatinine | Every three months until Month 15 | Concentration of serum creatinine |
Countries
France
Outcome results
None listed