Clinical trial NCT04561466: Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Official title: Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
PHASE3
Conditions: Safety Issues
Efficacy, Self
Keywords: leber herediatry optic neuropathy
befizal
Lead sponsor: Hôpital Necker-Enfants Malades
Sponsor class: OTHER
Collaborators: name: European Georges Pompidou Hospital
name: CLAIROP
First posted: 2020-09-23
Enrollment: 14
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Intervention model description: open
Primary purpose: TREATMENT

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

Eligibility 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

Primary outcomes:
measure: Evolution of best corrected farsight visual acuity (in LogMAR)
timeFrame: Month12
Secondary outcomes:
measure: Farsight best corrected visual acuity
timeFrame: Month 3
measure: Evolution of Farsight best corrected visual acuity
timeFrame: Month 6
measure: Evolution of Farsight best corrected visual acuity
timeFrame: Month 9
measure: Evolution of Farsight best corrected visual acuity
timeFrame: Month 15
measure: Evolution of Farsight best corrected visual acuity
timeFrame: Month 3
measure: Evolution of Farsight decimal best corrected visual acuity
timeFrame: Month 6
measure: Evolution of Farsight decimal best corrected visual acuity
timeFrame: Month 9
measure: Evolution of Farsight decimal best corrected visual acuity
timeFrame: Month 12
measure: Evolution of Farsight decimal best corrected visual acuity
timeFrame: Month 15
measure: Evolution of Nearsight visual acuity
timeFrame: Month 3
measure: Evolution of Nearsight visual acuity
timeFrame: Month 6
measure: Evolution of Nearsight visual acuity
timeFrame: Month 9
measure: Evolution of Nearsight visual acuity
timeFrame: Month 12
measure: Evolution of Nearsight visual acuity
timeFrame: Month 15
measure: Evolution of retinal nerve fibers layer Optical Coherent Tomography
timeFrame: Month 6
measure: Evolution of retinal nerve fibers layer Optical Coherent Tomography
timeFrame: Month 12
measure: automated visual field measurement
timeFrame: Month 3
measure: automated visual field measurement
timeFrame: Month 6
measure: automated visual field
timeFrame: Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0
measure: automated visual field measurement
timeFrame: Month 12
measure: automated visual field measurement
timeFrame: Month 15
measure: Manual visual field measurement
timeFrame: Month 3
measure: Manual visual field measurement
timeFrame: Month 6
measure: Manual visual field measurement
timeFrame: Month 9
measure: Manual visual field measurement
timeFrame: Month 12
measure: Manual visual field measurement
timeFrame: Month 15
measure: National Eye Institute Visual Function Questionnaire 25
timeFrame: Month 12
measure: Concentration of serum creatinine
timeFrame: Every three months until Month 15
measure: Concentration of high-density lipoprotein cholesterol
timeFrame: Every three months until Month 15
measure: Concentration of very low-density lipoprotein cholesterol
timeFrame: Every three months until Month 15
measure: Concentration of triglycerides
timeFrame: Every three months until Month 15
measure: Rate of Partial thromboplastin time
timeFrame: Every three months until Month 15
measure: Concentration of Aspartate aminotransférase
timeFrame: Every three months until Month 15
measure: Concentration of Alanine aminotransférase
timeFrame: Every three months until Month 15
measure: Concentration of lactate deshydrogenase
timeFrame: Every three months until Month 15
measure: Concentration of creatine kinase
timeFrame: Every three months until Month 15
Other outcomes:
Locations:
city: Paris
country: France
facility: HEGP Hospital
Study officials:
affiliation: Necker Hopsital
name: Dominique Bremond Gignac, MD PhD
role: STUDY_CHAIR
References: