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

Safety Issues Clinical Trial

— Béfinohl  

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04561466
• Other study ID #: beza16
• Status: Completed
• Phase: Phase 2/Phase 3
• Start date: March 26, 2019
• Est. completion date: March 10, 2023

Study information

• Verified date: September 2023
• Source: Hôpital Necker-Enfants Malades
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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

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

Recruitment information / eligibility

• Status: Completed
• Enrollment: 14
• Est. completion date: March 10, 2023
• Est. primary completion date: December 9, 2022
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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

Related Conditions & MeSH terms

• Efficacy, Self
• Optic Atrophy, Hereditary, Leber
• Optic Nerve Diseases
• Safety Issues

Intervention

Drug:
• Béfizal
600 mf befizal a day for one year

Locations

Country	Name	City	State
France	HEGP Hospital	Paris

Sponsors (3)

Lead Sponsor	Collaborator
Hôpital Necker-Enfants Malades	CLAIROP, European Georges Pompidou Hospital

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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	Month12
Secondary	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	Month 3
Secondary	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	Month 6
Secondary	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	Month 9
Secondary	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	Month 15
Secondary	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	Month 3
Secondary	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	Month 6
Secondary	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	Month 9
Secondary	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	Month 12
Secondary	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	Month 15
Secondary	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	Month 3
Secondary	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	Month 6
Secondary	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	Month 9
Secondary	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	Month 12
Secondary	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	Month 15
Secondary	Evolution of retinal nerve fibers layer Optical Coherent Tomography	physiological parameter : Optical Coherent Tomography	Month 6
Secondary	Evolution of retinal nerve fibers layer Optical Coherent Tomography	physiological parameter : Optical Coherent Tomography	Month 12
Secondary	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)	Month 3
Secondary	automated visual field measurement	physiological parameter : automated visual field	Month 6
Secondary	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)	Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0
Secondary	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)	Month 12
Secondary	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)	Month 15
Secondary	Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 3
Secondary	Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 6
Secondary	Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 9
Secondary	Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 12
Secondary	Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 15
Secondary	National Eye Institute Visual Function Questionnaire 25	Questionnaire	Month 12
Secondary	Concentration of serum creatinine	Concentration of serum creatinine	Every three months until Month 15
Secondary	Concentration of high-density lipoprotein cholesterol	Blood test	Every three months until Month 15
Secondary	Concentration of very low-density lipoprotein cholesterol	Blood test	Every three months until Month 15
Secondary	Concentration of triglycerides	Blood test	Every three months until Month 15
Secondary	Rate of Partial thromboplastin time	Blood test	Every three months until Month 15
Secondary	Concentration of Aspartate aminotransférase	Blood test	Every three months until Month 15
Secondary	Concentration of Alanine aminotransférase	Blood test	Every three months until Month 15
Secondary	Concentration of lactate deshydrogenase	Blood test	Every three months until Month 15
Secondary	Concentration of creatine kinase	Blood test	Every three months until Month 15