NCT04561466

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

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Mar 2019

Typical duration for phase_2

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

First Submitted

Initial submission to the registry

March 14, 2019

Completed
12 days until next milestone

Study Start

First participant enrolled

March 26, 2019

Completed
1.5 years until next milestone

First Posted

Study publicly available on registry

September 23, 2020

Completed
2.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 9, 2022

Completed
3 months until next milestone

Study Completion

Last participant's last visit for all outcomes

March 10, 2023

Completed
Last Updated

September 22, 2023

Status Verified

September 1, 2023

Enrollment Period

3.7 years

First QC Date

March 14, 2019

Last Update Submit

September 21, 2023

Conditions

Safety IssuesEfficacy, Self

Keywords

leber herediatry optic neuropathybefizal

Outcome Measures

Primary Outcomes (1)

  • 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 Outcomes (36)

  • Farsight best corrected visual acuity

    Month 3

  • Evolution of Farsight best corrected visual acuity

    Month 6

  • Evolution of Farsight best corrected visual acuity

    Month 9

  • Evolution of Farsight best corrected visual acuity

    Month 15

  • Evolution of Farsight best corrected visual acuity

    Month 3

  • +31 more secondary outcomes

Study Arms (1)

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

Drug: Béfizal

Interventions

BéfizalDRUG

600 mf befizal a day for one year

treatment group

Eligibility Criteria

Age18 Years+
Sexall
Healthy VolunteersNo
Age GroupsAdult (18-64), Older Adult (65+)

You may qualify if:

  • 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

You may not qualify if:

  • \* 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

HEGP Hospital

Paris, 75015, France

Location

MeSH Terms

Interventions

Bezafibrate

Intervention Hierarchy (Ancestors)

BenzamidesAmidesOrganic ChemicalsFibric AcidsIsobutyratesButyratesAcids, AcyclicCarboxylic AcidsBenzoatesAcids, CarbocyclicChlorobenzoatesPhenyl EthersEthersBenzene DerivativesHydrocarbons, AromaticHydrocarbons, CyclicHydrocarbonsPhenols

Study Officials

  • Dominique Bremond Gignac, MD PhD

    Necker Hopsital

    STUDY CHAIR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Model Details: open
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal investigator

Study Record Dates

First Submitted

March 14, 2019

First Posted

September 23, 2020

Study Start

March 26, 2019

Primary Completion

December 9, 2022

Study Completion

March 10, 2023

Last Updated

September 22, 2023

Record last verified: 2023-09

Locations

FR(1)