Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Trial Snapshot
- Phase
- Phase 2
- Status
- Completed
- Sponsor
- Enrollment
- 14
- Locations
- 1
- Primary Endpoint
- Evolution of best corrected farsight visual acuity (in LogMAR)
Study Overview
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
Study Design
- Study Type
- Interventional
- Allocation
- Na
- Intervention Model
- Single Group
- Primary Purpose
- Treatment
- Masking
- None
Eligibility Criteria
- Ages
- 18 Years to — (Adult, Older Adult)
- Sex
- All
- Accepts 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
Arms & Interventions
treatment group
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
Intervention: Béfizal (Drug)
Outcomes
Primary Outcomes
Evolution of best corrected farsight visual acuity (in LogMAR)
Time Frame: 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 Outcomes
- Evolution of Farsight decimal best corrected visual acuity(Month 15)
- Evolution of retinal nerve fibers layer Optical Coherent Tomography(Month 12)
- Concentration of serum creatinine(Every three months until Month 15)
- Rate of Partial thromboplastin time(Every three months until Month 15)
- Farsight best corrected visual acuity(Month 3)
- automated visual field(Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0)
- Evolution of Farsight best corrected visual acuity(Month 3)
- Evolution of Nearsight visual acuity(Month 15)
- Concentration of Alanine aminotransférase(Every three months until Month 15)
- automated visual field measurement(Month 15)
- Concentration of lactate deshydrogenase(Every three months until Month 15)
- Manual visual field measurement(Month 15)
- National Eye Institute Visual Function Questionnaire 25(Month 12)
- Concentration of high-density lipoprotein cholesterol(Every three months until Month 15)
- Concentration of very low-density lipoprotein cholesterol(Every three months until Month 15)
- Concentration of triglycerides(Every three months until Month 15)
- Concentration of Aspartate aminotransférase(Every three months until Month 15)
- Concentration of creatine kinase(Every three months until Month 15)
Investigators
Christophe Orssaud
Principal investigator
Hôpital Necker-Enfants Malades