Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy (Béfinohl)
Primary Purpose
Safety Issues, Efficacy, Self
Status
Completed
Phase
Phase 2
Locations
France
Study Type
Interventional
Intervention
Béfizal
Sponsored by
About this trial
This is an interventional treatment trial for Safety Issues focused on measuring leber herediatry optic neuropathy, befizal
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
Sites / Locations
- HEGP Hospital
Arms of the Study
Arm 1
Arm Type
Experimental
Arm Label
treatment group
Arm Description
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
Outcomes
Primary Outcome Measures
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
Secondary Outcome Measures
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Evolution of retinal nerve fibers layer Optical Coherent Tomography
physiological parameter : Optical Coherent Tomography
Evolution of retinal nerve fibers layer Optical Coherent Tomography
physiological parameter : Optical Coherent Tomography
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)
automated visual field measurement
physiological parameter : automated visual field
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)
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)
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)
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
National Eye Institute Visual Function Questionnaire 25
Questionnaire
Concentration of serum creatinine
Concentration of serum creatinine
Concentration of high-density lipoprotein cholesterol
Blood test
Concentration of very low-density lipoprotein cholesterol
Blood test
Concentration of triglycerides
Blood test
Rate of Partial thromboplastin time
Blood test
Concentration of Aspartate aminotransférase
Blood test
Concentration of Alanine aminotransférase
Blood test
Concentration of lactate deshydrogenase
Blood test
Concentration of creatine kinase
Blood test
Full Information
NCT ID
NCT04561466
First Posted
March 14, 2019
Last Updated
September 21, 2023
Sponsor
Hôpital Necker-Enfants Malades
Collaborators
European Georges Pompidou Hospital, CLAIROP
1. Study Identification
Unique Protocol Identification Number
NCT04561466
Brief Title
Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Acronym
Béfinohl
Official Title
Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Study Type
Interventional
2. Study Status
Record Verification Date
September 2023
Overall Recruitment Status
Completed
Study Start Date
March 26, 2019 (Actual)
Primary Completion Date
December 9, 2022 (Actual)
Study Completion Date
March 10, 2023 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Hôpital Necker-Enfants Malades
Collaborators
European Georges Pompidou Hospital, CLAIROP
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
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
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Safety Issues, Efficacy, Self
Keywords
leber herediatry optic neuropathy, befizal
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2, Phase 3
Interventional Study Model
Single Group Assignment
Model Description
open
Masking
None (Open Label)
Allocation
N/A
Enrollment
14 (Actual)
8. Arms, Groups, and Interventions
Arm Title
treatment group
Arm Type
Experimental
Arm Description
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 Type
Drug
Intervention Name(s)
Béfizal
Intervention Description
600 mf befizal a day for one year
Primary Outcome Measure Information:
Title
Evolution of best corrected farsight visual acuity (in LogMAR)
Description
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 Outcome Measure Information:
Title
Farsight best corrected visual acuity
Description
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
Title
Evolution of Farsight best corrected visual acuity
Description
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
Title
Evolution of Farsight best corrected visual acuity
Description
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
Title
Evolution of Farsight best corrected visual acuity
Description
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
Title
Evolution of Farsight best corrected visual acuity
Description
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
Time Frame
Month 3
Title
Evolution of Farsight decimal best corrected visual acuity
Description
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
Title
Evolution of Farsight decimal best corrected visual acuity
Description
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
Title
Evolution of Farsight decimal best corrected visual acuity
Description
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
Title
Evolution of Farsight decimal best corrected visual acuity
Description
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
Title
Evolution of Nearsight visual acuity
Description
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
Title
Evolution of Nearsight visual acuity
Description
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
Title
Evolution of Nearsight visual acuity
Description
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
Title
Evolution of Nearsight visual acuity
Description
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
Title
Evolution of Nearsight visual acuity
Description
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
Title
Evolution of retinal nerve fibers layer Optical Coherent Tomography
Description
physiological parameter : Optical Coherent Tomography
Time Frame
Month 6
Title
Evolution of retinal nerve fibers layer Optical Coherent Tomography
Description
physiological parameter : Optical Coherent Tomography
Time Frame
Month 12
Title
automated visual field measurement
Description
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
Title
automated visual field measurement
Description
physiological parameter : automated visual field
Time Frame
Month 6
Title
automated visual field
Description
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
Title
automated visual field measurement
Description
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
Title
automated visual field measurement
Description
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
Title
Manual visual field measurement
Description
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Time Frame
Month 3
Title
Manual visual field measurement
Description
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Time Frame
Month 6
Title
Manual visual field measurement
Description
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Time Frame
Month 9
Title
Manual visual field measurement
Description
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Time Frame
Month 12
Title
Manual visual field measurement
Description
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Time Frame
Month 15
Title
National Eye Institute Visual Function Questionnaire 25
Description
Questionnaire
Time Frame
Month 12
Title
Concentration of serum creatinine
Description
Concentration of serum creatinine
Time Frame
Every three months until Month 15
Title
Concentration of high-density lipoprotein cholesterol
Description
Blood test
Time Frame
Every three months until Month 15
Title
Concentration of very low-density lipoprotein cholesterol
Description
Blood test
Time Frame
Every three months until Month 15
Title
Concentration of triglycerides
Description
Blood test
Time Frame
Every three months until Month 15
Title
Rate of Partial thromboplastin time
Description
Blood test
Time Frame
Every three months until Month 15
Title
Concentration of Aspartate aminotransférase
Description
Blood test
Time Frame
Every three months until Month 15
Title
Concentration of Alanine aminotransférase
Description
Blood test
Time Frame
Every three months until Month 15
Title
Concentration of lactate deshydrogenase
Description
Blood test
Time Frame
Every three months until Month 15
Title
Concentration of creatine kinase
Description
Blood test
Time Frame
Every three months until Month 15
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Dominique Bremond Gignac, MD PhD
Organizational Affiliation
Necker Hopsital
Official's Role
Study Chair
Facility Information:
Facility Name
HEGP Hospital
City
Paris
ZIP/Postal Code
75015
Country
France
12. IPD Sharing Statement
Plan to Share IPD
Undecided
IPD Sharing Plan Description
Undecided
Learn more about this trial
Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
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