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

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ClinicalTrials.gov Identifier: NCT04561466
Recruitment Status : Recruiting
First Posted : September 23, 2020
Last Update Posted : February 12, 2021
Sponsor:
Collaborators:
European Georges Pompidou Hospital
CLAIROP
Information provided by (Responsible Party):
Christophe Orssaud, Hôpital Necker-Enfants Malades

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

Condition or disease Intervention/treatment Phase
Safety Issues Efficacy, Self Drug: Béfizal Phase 2 Phase 3

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

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 14 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Intervention Model Description: open
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Actual Study Start Date : March 26, 2019
Estimated Primary Completion Date : December 9, 2022
Estimated Study Completion Date : March 10, 2023


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




Primary Outcome Measures :
  1. 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 Outcome Measures :
  1. Farsight best corrected visual acuity [ Time Frame: Month 3 ]
    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

  2. Evolution of Farsight best corrected visual acuity [ Time Frame: Month 6 ]
    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

  3. Evolution of Farsight best corrected visual acuity [ Time Frame: Month 9 ]
    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

  4. Evolution of Farsight best corrected visual acuity [ Time Frame: Month 15 ]
    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

  5. Evolution of Farsight best corrected visual acuity [ Time Frame: Month 3 ]
    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

  6. Evolution of Farsight decimal best corrected visual acuity [ Time Frame: Month 6 ]
    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

  7. Evolution of Farsight decimal best corrected visual acuity [ Time Frame: Month 9 ]
    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

  8. Evolution of Farsight decimal best corrected visual acuity [ Time Frame: Month 12 ]
    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

  9. Evolution of Farsight decimal best corrected visual acuity [ Time Frame: Month 15 ]
    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

  10. Evolution of Nearsight visual acuity [ Time Frame: Month 3 ]
    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

  11. Evolution of Nearsight visual acuity [ Time Frame: Month 6 ]
    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

  12. Evolution of Nearsight visual acuity [ Time Frame: Month 9 ]
    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

  13. Evolution of Nearsight visual acuity [ Time Frame: Month 12 ]
    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

  14. Evolution of Nearsight visual acuity [ Time Frame: Month 15 ]
    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

  15. Evolution of retinal nerve fibers layer Optical Coherent Tomography [ Time Frame: Month 6 ]
    physiological parameter : Optical Coherent Tomography

  16. Evolution of retinal nerve fibers layer Optical Coherent Tomography [ Time Frame: Month 12 ]
    physiological parameter : Optical Coherent Tomography

  17. automated visual field measurement [ Time Frame: Month 3 ]
    physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)

  18. automated visual field measurement [ Time Frame: Month 6 ]
    physiological parameter : automated visual field

  19. automated visual field [ Time Frame: Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0 ]
    physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)

  20. automated visual field measurement [ Time Frame: Month 12 ]
    physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)

  21. automated visual field measurement [ Time Frame: Month 15 ]
    physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)

  22. Manual visual field measurement [ Time Frame: Month 3 ]
    physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

  23. Manual visual field measurement [ Time Frame: Month 6 ]
    physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

  24. Manual visual field measurement [ Time Frame: Month 9 ]
    physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

  25. Manual visual field measurement [ Time Frame: Month 12 ]
    physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

  26. Manual visual field measurement [ Time Frame: Month 15 ]
    physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1

  27. National Eye Institute Visual Function Questionnaire 25 [ Time Frame: Month 12 ]
    Questionnaire

  28. Concentration of serum creatinine [ Time Frame: Every three months until Month 15 ]
    Concentration of serum creatinine

  29. Concentration of high-density lipoprotein cholesterol [ Time Frame: Every three months until Month 15 ]
    Blood test

  30. Concentration of very low-density lipoprotein cholesterol [ Time Frame: Every three months until Month 15 ]
    Blood test

  31. Concentration of triglycerides [ Time Frame: Every three months until Month 15 ]
    Blood test

  32. Rate of Partial thromboplastin time [ Time Frame: Every three months until Month 15 ]
    Blood test

  33. Concentration of Aspartate aminotransférase [ Time Frame: Every three months until Month 15 ]
    Blood test

  34. Concentration of Alanine aminotransférase [ Time Frame: Every three months until Month 15 ]
    Blood test

  35. Concentration of lactate deshydrogenase [ Time Frame: Every three months until Month 15 ]
    Blood test

  36. Concentration of creatine kinase [ Time Frame: Every three months until Month 15 ]
    Blood test



Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04561466


Contacts
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Contact: christophe Orssaud, MD 33 1 56 09 34 66 christophe.orssaud@aphp.fr

Locations
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France
HEGP Hospital Recruiting
Paris, France, 75015
Contact: Christophe ORSSAUD, MD    33 1 56 09 34 98    christophe.orssaud@aphp.fr   
Contact: Dominique Bremond Gignac, MD PhD    33 1 44 49 45 02    dominique.bremond@aphp.fr   
Sponsors and Collaborators
Hôpital Necker-Enfants Malades
European Georges Pompidou Hospital
CLAIROP
Investigators
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Study Chair: Dominique Bremond Gignac, MD PhD Necker Hopsital
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Responsible Party: Christophe Orssaud, Principal investigator, Hôpital Necker-Enfants Malades
ClinicalTrials.gov Identifier: NCT04561466    
Other Study ID Numbers: beza16
First Posted: September 23, 2020    Key Record Dates
Last Update Posted: February 12, 2021
Last Verified: February 2021
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Plan Description: Undecided

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Christophe Orssaud, Hôpital Necker-Enfants Malades:
leber herediatry optic neuropathy
befizal
Additional relevant MeSH terms:
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Optic Nerve Diseases
Optic Atrophy, Hereditary, Leber
Cranial Nerve Diseases
Nervous System Diseases
Eye Diseases
Optic Atrophies, Hereditary
Optic Atrophy
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Eye Diseases, Hereditary
Genetic Diseases, Inborn
Mitochondrial Diseases
Metabolic Diseases
Bezafibrate
Hypolipidemic Agents
Antimetabolites
Molecular Mechanisms of Pharmacological Action
Lipid Regulating Agents