---
title: Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
nct_id: NCT04561466
overall_status: COMPLETED
phase: PHASE2, PHASE3
sponsor: Hôpital Necker-Enfants Malades
study_type: INTERVENTIONAL
primary_condition: Safety Issues
countries: France
canonical_url: "https://parkinsonspathways.com/agent/trials/NCT04561466.md"
clinicaltrials_gov: "https://clinicaltrials.gov/study/NCT04561466"
ct_last_update_post_date: 2023-09-22
last_seen_at: "2026-05-12T07:08:26.085Z"
source: ClinicalTrials.gov (mirrored, no enrichment)
---
# Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy

**Official Title:** Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy

**NCT ID:** [NCT04561466](https://clinicaltrials.gov/study/NCT04561466)

## Key Facts

- **Status:** COMPLETED
- **Phase:** PHASE2, PHASE3
- **Study Type:** INTERVENTIONAL
- **Target Enrollment:** 14
- **Lead Sponsor:** Hôpital Necker-Enfants Malades
- **Collaborators:** European Georges Pompidou Hospital, CLAIROP
- **Conditions:** Safety Issues, Efficacy, Self
- **Start Date:** 2019-03-26
- **Completion Date:** 2023-03-10
- **CT.gov Last Update:** 2023-09-22

## 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

## 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

## Eligibility

- **Minimum age:** 18 Years
- **Sex:** ALL
- **Healthy Volunteers:** No

```
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
```

## Arms

- **treatment group** (EXPERIMENTAL) — 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

## Interventions

- **Béfizal** (DRUG) — 600 mf befizal a day for one year

## Primary Outcomes

- **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

- **Farsight best corrected visual acuity** _(time frame: Month 3)_
- **Evolution of Farsight best corrected visual acuity** _(time frame: Month 6)_
- **Evolution of Farsight best corrected visual acuity** _(time frame: Month 9)_
- **Evolution of Farsight best corrected visual acuity** _(time frame: Month 15)_
- **Evolution of Farsight best corrected visual acuity** _(time frame: Month 3)_
- **Evolution of Farsight decimal best corrected visual acuity** _(time frame: Month 6)_
- **Evolution of Farsight decimal best corrected visual acuity** _(time frame: Month 9)_
- **Evolution of Farsight decimal best corrected visual acuity** _(time frame: Month 12)_
- **Evolution of Farsight decimal best corrected visual acuity** _(time frame: Month 15)_
- **Evolution of Nearsight visual acuity** _(time frame: Month 3)_
- **Evolution of Nearsight visual acuity** _(time frame: Month 6)_
- **Evolution of Nearsight visual acuity** _(time frame: Month 9)_
- **Evolution of Nearsight visual acuity** _(time frame: Month 12)_
- **Evolution of Nearsight visual acuity** _(time frame: Month 15)_
- **Evolution of retinal nerve fibers layer Optical Coherent Tomography** _(time frame: Month 6)_
- **Evolution of retinal nerve fibers layer Optical Coherent Tomography** _(time frame: Month 12)_
- **automated visual field measurement** _(time frame: Month 3)_
- **automated visual field measurement** _(time frame: Month 6)_
- **automated visual field** _(time frame: Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0)_
- **automated visual field measurement** _(time frame: Month 12)_
- **automated visual field measurement** _(time frame: Month 15)_
- **Manual visual field measurement** _(time frame: Month 3)_
- **Manual visual field measurement** _(time frame: Month 6)_
- **Manual visual field measurement** _(time frame: Month 9)_
- **Manual visual field measurement** _(time frame: Month 12)_
- **Manual visual field measurement** _(time frame: Month 15)_
- **National Eye Institute Visual Function Questionnaire 25** _(time frame: Month 12)_
- **Concentration of serum creatinine** _(time frame: Every three months until Month 15)_
- **Concentration of high-density lipoprotein cholesterol** _(time frame: Every three months until Month 15)_
- **Concentration of very low-density lipoprotein cholesterol** _(time frame: Every three months until Month 15)_
- **Concentration of triglycerides** _(time frame: Every three months until Month 15)_
- **Rate of Partial thromboplastin time** _(time frame: Every three months until Month 15)_
- **Concentration of Aspartate aminotransférase** _(time frame: Every three months until Month 15)_
- **Concentration of Alanine aminotransférase** _(time frame: Every three months until Month 15)_
- **Concentration of lactate deshydrogenase** _(time frame: Every three months until Month 15)_
- **Concentration of creatine kinase** _(time frame: Every three months until Month 15)_

## Locations (1)

- HEGP Hospital, Paris, France

## Recent Field Changes (last 30 days)

- `status.overallStatus` — added _(2026-05-12)_
- `status.primaryCompletionDate` — added _(2026-05-12)_
- `status.completionDate` — added _(2026-05-12)_
- `status.lastUpdatePostDate` — added _(2026-05-12)_
- `design.phases` — added _(2026-05-12)_
- `design.enrollmentCount` — added _(2026-05-12)_
- `eligibility.criteria` — added _(2026-05-12)_
- `eligibility.minAge` — added _(2026-05-12)_
- `eligibility.sex` — added _(2026-05-12)_
- `outcomes.primary` — added _(2026-05-12)_
- `outcomes.secondary` — added _(2026-05-12)_
- `armsInterventions.arms` — added _(2026-05-12)_
- `armsInterventions.interventions` — added _(2026-05-12)_
- `sponsor.lead` — added _(2026-05-12)_
- `sponsor.collaborators` — added _(2026-05-12)_
- `results.hasResults` — added _(2026-05-12)_
- `locations.hegp hospital|paris||france` — added _(2026-05-12)_

---

*Canonical: https://parkinsonspathways.com/agent/trials/NCT04561466.md*  
*Source data (authoritative): https://clinicaltrials.gov/study/NCT04561466*  
*This page is a raw mirror with no AI summary, no editorial enrichment, and no Parkinson's-specific filtering.*