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

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

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

Study Overview

• Status: Completed
• Conditions: Safety Issues; Efficacy, Self
• Intervention / Treatment: Drug: Béfizal

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 Type: Interventional
• Enrollment (Actual): 14
• Phase: Phase 2; Phase 3

Contacts and Locations

• Study Contact: Name: christophe Orssaud, MD; Phone Number: 33 1 56 09 34 66; Email: christophe.orssaud@aphp.fr

Study Locations

• France
  • Paris, France, 75015
    • HEGP Hospital

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No

Description

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

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: 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

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

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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
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
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
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
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
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
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
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
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
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
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
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
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
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
Evolution of retinal nerve fibers layer Optical Coherent Tomography	physiological parameter : Optical Coherent Tomography	Month 6
Evolution of retinal nerve fibers layer Optical Coherent Tomography	physiological parameter : Optical Coherent Tomography	Month 12
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
automated visual field measurement	physiological parameter : automated visual field	Month 6
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
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
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
Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 3
Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 6
Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 9
Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 12
Manual visual field measurement	physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1	Month 15
National Eye Institute Visual Function Questionnaire 25	Questionnaire	Month 12
Concentration of serum creatinine	Concentration of serum creatinine	Every three months until Month 15
Concentration of high-density lipoprotein cholesterol	Blood test	Every three months until Month 15
Concentration of very low-density lipoprotein cholesterol	Blood test	Every three months until Month 15
Concentration of triglycerides	Blood test	Every three months until Month 15
Rate of Partial thromboplastin time	Blood test	Every three months until Month 15
Concentration of Aspartate aminotransférase	Blood test	Every three months until Month 15
Concentration of Alanine aminotransférase	Blood test	Every three months until Month 15
Concentration of lactate deshydrogenase	Blood test	Every three months until Month 15
Concentration of creatine kinase	Blood test	Every three months until Month 15

Collaborators and Investigators

• Sponsor: Hôpital Necker-Enfants Malades
• Collaborators: European Georges Pompidou Hospital; CLAIROP
• Investigators: Study Chair: Dominique Bremond Gignac, MD PhD, Necker Hopsital

Study record dates

Study Major Dates

• Study Start (Actual): March 26, 2019
• Primary Completion (Actual): December 9, 2022
• Study Completion (Actual): March 10, 2023

Study Registration Dates

• First Submitted: March 14, 2019
• First Submitted That Met QC Criteria: September 17, 2020
• First Posted (Actual): September 23, 2020

Study Record Updates

• Last Update Posted (Actual): September 22, 2023
• Last Update Submitted That Met QC Criteria: September 21, 2023
• Last Verified: September 1, 2023

More Information

Terms related to this study

• Keywords: leber herediatry optic neuropathy; befizal
• Additional Relevant MeSH Terms: Metabolic Diseases; Nervous System Diseases; Eye Diseases; Genetic Diseases, Inborn; Neurodegenerative Diseases; Eye Diseases, Hereditary; Heredodegenerative Disorders, Nervous System; Cranial Nerve Diseases; Optic Atrophies, Hereditary; Optic Atrophy; Mitochondrial Diseases; Optic Nerve Diseases; Optic Atrophy, Hereditary, Leber; Molecular Mechanisms of Pharmacological Action; Antimetabolites; Hypolipidemic Agents; Lipid Regulating Agents; Bezafibrate
• Other Study ID Numbers: beza16

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: UNDECIDED
• IPD Plan Description: Undecided

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No