Safety IssuesPhase 2/3BéfinohlNCT04561466
Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
A Phase 2/3 study testing Béfizal in people with Safety Issues and Efficacy, Self, run by Hôpital Necker-Enfants Malades at 1 site in France.
Trial 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 Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Safety Issues, Efficacy, Self
- What is being tested?
- Béfizal
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteFrance
- Study dates
- 26 Mar 2019to 10 Mar 2023
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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 cannot join
- * 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);
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- 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 locations and contacts
1 site in 1 country.
About this study
Official title: Study of Efficacy 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
Read the 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
- Conditions
- Safety Issues, Efficacy, Self
- Being tested
- Béfizal
- Keywords
- leber herediatry optic neuropathy, befizal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BéfizalDrug
600 mf befizal a day for one year
| Study group | Role | What they receive |
|---|---|---|
| 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 Receives: Drug: Béfizal |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: Month12
Secondary outcomes (36)
Farsight best corrected visual acuity
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
Measured: Month 3
Evolution of Farsight best corrected visual acuity
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
Measured: Month 6
Evolution of Farsight best corrected visual acuity
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
Measured: Month 9
Evolution of Farsight best corrected visual acuity
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
Measured: Month 15
Evolution of Farsight best corrected visual acuity
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
Measured: Month 3
Evolution of Farsight decimal best corrected visual acuity
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
Measured: Month 6
Evolution of Farsight decimal best corrected visual acuity
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
Measured: Month 9
Evolution of Farsight decimal best corrected visual acuity
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
Measured: Month 12
Evolution of Farsight decimal best corrected visual acuity
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
Measured: Month 15
Evolution of Nearsight visual acuity
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
Measured: Month 3
Evolution of Nearsight visual acuity
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
Measured: Month 6
Evolution of Nearsight visual acuity
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
Measured: Month 9
Evolution of Nearsight visual acuity
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
Measured: Month 12
Evolution of Nearsight visual acuity
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
Measured: Month 15
Evolution of retinal nerve fibers layer Optical Coherent Tomography
physiological parameter : Optical Coherent Tomography
Measured: Month 6
Evolution of retinal nerve fibers layer Optical Coherent Tomography
physiological parameter : Optical Coherent Tomography
Measured: Month 12
automated visual field measurement
physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)
Measured: Month 3
automated visual field measurement
physiological parameter : automated visual field
Measured: Month 6
automated visual field
physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)
Measured: Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0
automated visual field measurement
physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)
Measured: Month 12
automated visual field measurement
physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)
Measured: Month 15
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Measured: Month 3
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Measured: Month 6
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Measured: Month 9
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Measured: Month 12
Manual visual field measurement
physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1
Measured: Month 15
National Eye Institute Visual Function Questionnaire 25
Questionnaire
Measured: Month 12
Concentration of serum creatinine
Concentration of serum creatinine
Measured: Every three months until Month 15
Concentration of high-density lipoprotein cholesterol
Blood test
Measured: Every three months until Month 15
Concentration of very low-density lipoprotein cholesterol
Blood test
Measured: Every three months until Month 15
Concentration of triglycerides
Blood test
Measured: Every three months until Month 15
Rate of Partial thromboplastin time
Blood test
Measured: Every three months until Month 15
Concentration of Aspartate aminotransférase
Blood test
Measured: Every three months until Month 15
Concentration of Alanine aminotransférase
Blood test
Measured: Every three months until Month 15
Concentration of lactate deshydrogenase
Blood test
Measured: Every three months until Month 15
Concentration of creatine kinase
Blood test
Measured: Every three months until Month 15
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2020
- Study start26 Mar 2019
- Primary completion9 Dec 2022
- Study completion10 Mar 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Safety IssuesStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Model description
- open
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Hôpital Necker-Enfants Malades
- Sponsor type
- University, hospital or other
- Collaborators
- European Georges Pompidou Hospital, CLAIROP
- Responsible party
- Principal investigator, Christophe Orssaud, Hôpital Necker-Enfants Malades
- Investigators
- Dominique Bremond Gignac, MD PhD, Study chair, Necker Hopsital
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04561466
- Sponsor's protocol ID
- beza16
- Acronym
- Béfinohl
- Status last verified
- Sep 2023
- First submitted
- 14 Mar 2019
- Last updated
- 22 Sep 2023
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided: Undecided
Study at a glance
From the registry record, last updated by the sponsor on 22 Sep 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
- ClinicalTrials.gov ID
- NCT04561466
- Acronym / sponsor ID
- Béfinohl / beza16
- Conditions
- Safety Issues, Efficacy, Self
- Keywords
- leber herediatry optic neuropathy, befizal
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Hôpital Necker-Enfants MaladesUniversity, hospital or other
- Collaborators
- European Georges Pompidou Hospital, CLAIROP
- Study start
- 26 Mar 2019 (actual)
- Primary completion
- 9 Dec 2022 (actual)
- Study completion
- 10 Mar 2023 (actual)
- First posted
- 23 Sep 2020 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
- Model description
- open
| Arm | Type | Receives |
|---|---|---|
| treatment group | Experimental | Drug: Béfizal14 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 |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (1 listed)
- 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
Key exclusion criteria (12 listed)
- * Any optic neuropathy for which the diagnosis of LHON is not formally confirmed or genetically proven;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Béfizal600 mf befizal a day for one year | Drug | treatment group |
Primary outcome
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
36 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Sep 2023.
- Individual participant dataSharing planned: Undecided. Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Dominique Bremond Gignac, MD PhD study chair
Necker Hopsital
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing Béfizal in people with Safety Issues and Efficacy, Self, run by Hôpital Necker-Enfants Malades at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Sep 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Evolution of best corrected farsight visual acuity (in LogMAR) (time frame: Month12). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Sep 2023.