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Completed

NCT Number: NCT04561466

Trial 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

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Key information

Conditions

Safety Issues Efficacy, Self

Age range

18 year and older

Sex eligibility

All sexes

Study type

Interventional

Phase

Phase 2 / Phase 3

Primary location

HEGP Hospital

Paris, 75015, France

About this study

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

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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

Treatment and study plan

Béfizal

Drug

600 mf befizal a day for one year

Primary outcomes

  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 outcomes

  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

Sponsors and collaborators

Lead sponsor

Hôpital Necker-Enfants Malades

Other

Collaborators

  • CLAIROP
  • European Georges Pompidou Hospital

Registry information

Official study title

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

Acronym: Béfinohl

Important dates

Study start
2019
Primary completion
2022
Study completion
2023
First posted
Sep 23, 2020
Registry last updated
Sep 22, 2023

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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