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

Hôpital Necker-Enfants Malades

Status and phase: Completed; Phase 3; Phase 2

Conditions: Safety Issues; Efficacy, Self

Treatments: Drug: Béfizal

Study type: Interventional

Funder types: Other

Identifiers

NCT04561466

beza16

Details and patient eligibility

About

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

Full 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

Enrollment: 14 patients

Sex: All

Ages: 18+ years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: N/A

Interventional model: Single Group Assignment

Masking: None (Open label)

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14 participants in 1 patient group

treatment group

Experimental group

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

Treatment:

Drug: Béfizal