Ignite Creation Date:
2025-12-24 @ 4:01 PM
Ignite Modification Date:
2026-01-01 @ 8:32 PM
Study NCT ID:
NCT04561466
Status:
COMPLETED
Last Update Posted:
2023-09-22
First Post:
2019-03-14
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'interventionBrowseModule': {'meshes': [{'id': 'D001629', 'term': 'Bezafibrate'}], 'ancestors': [{'id': 'D001549', 'term': 'Benzamides'}, {'id': 'D000577', 'term': 'Amides'}, {'id': 'D009930', 'term': 'Organic Chemicals'}, {'id': 'D058607', 'term': 'Fibric Acids'}, {'id': 'D058610', 'term': 'Isobutyrates'}, {'id': 'D002087', 'term': 'Butyrates'}, {'id': 'D000144', 'term': 'Acids, Acyclic'}, {'id': 'D002264', 'term': 'Carboxylic Acids'}, {'id': 'D001565', 'term': 'Benzoates'}, {'id': 'D000146', 'term': 'Acids, Carbocyclic'}, {'id': 'D002723', 'term': 'Chlorobenzoates'}, {'id': 'D010647', 'term': 'Phenyl Ethers'}, {'id': 'D004987', 'term': 'Ethers'}, {'id': 'D001555', 'term': 'Benzene Derivatives'}, {'id': 'D006841', 'term': 'Hydrocarbons, Aromatic'}, {'id': 'D006844', 'term': 'Hydrocarbons, Cyclic'}, {'id': 'D006838', 'term': 'Hydrocarbons'}, {'id': 'D010636', 'term': 'Phenols'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP', 'interventionModelDescription': 'open'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 14}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2019-03-26', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-09', 'completionDateStruct': {'date': '2023-03-10', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2023-09-21', 'studyFirstSubmitDate': '2019-03-14', 'studyFirstSubmitQcDate': '2020-09-17', 'lastUpdatePostDateStruct': {'date': '2023-09-22', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2020-09-23', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2022-12-09', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Evolution of best corrected farsight visual acuity (in LogMAR)', 'timeFrame': 'Month12', 'description': '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'}], 'secondaryOutcomes': [{'measure': 'Farsight best corrected visual acuity', 'timeFrame': 'Month 3', 'description': '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'}, {'measure': 'Evolution of Farsight best corrected visual acuity', 'timeFrame': 'Month 6', 'description': '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'}, {'measure': 'Evolution of Farsight best corrected visual acuity', 'timeFrame': 'Month 9', 'description': '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'}, {'measure': 'Evolution of Farsight best corrected visual acuity', 'timeFrame': 'Month 15', 'description': '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'}, {'measure': 'Evolution of Farsight best corrected visual acuity', 'timeFrame': 'Month 3', 'description': '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'}, {'measure': 'Evolution of Farsight decimal best corrected visual acuity', 'timeFrame': 'Month 6', 'description': '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'}, {'measure': 'Evolution of Farsight decimal best corrected visual acuity', 'timeFrame': 'Month 9', 'description': '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'}, {'measure': 'Evolution of Farsight decimal best corrected visual acuity', 'timeFrame': 'Month 12', 'description': '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'}, {'measure': 'Evolution of Farsight decimal best corrected visual acuity', 'timeFrame': 'Month 15', 'description': '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'}, {'measure': 'Evolution of Nearsight visual acuity', 'timeFrame': 'Month 3', 'description': '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'}, {'measure': 'Evolution of Nearsight visual acuity', 'timeFrame': 'Month 6', 'description': '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'}, {'measure': 'Evolution of Nearsight visual acuity', 'timeFrame': 'Month 9', 'description': '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'}, {'measure': 'Evolution of Nearsight visual acuity', 'timeFrame': 'Month 12', 'description': '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'}, {'measure': 'Evolution of Nearsight visual acuity', 'timeFrame': 'Month 15', 'description': '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'}, {'measure': 'Evolution of retinal nerve fibers layer Optical Coherent Tomography', 'timeFrame': 'Month 6', 'description': 'physiological parameter : Optical Coherent Tomography'}, {'measure': 'Evolution of retinal nerve fibers layer Optical Coherent Tomography', 'timeFrame': 'Month 12', 'description': 'physiological parameter : Optical Coherent Tomography'}, {'measure': 'automated visual field measurement', 'timeFrame': 'Month 3', 'description': 'physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)'}, {'measure': 'automated visual field measurement', 'timeFrame': 'Month 6', 'description': 'physiological parameter : automated visual field'}, {'measure': 'automated visual field', 'timeFrame': 'Month 9 of a treatment with BEFIZAL® 200mg (ARROW GENERIQUES) compare to Month 0', 'description': 'physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)'}, {'measure': 'automated visual field measurement', 'timeFrame': 'Month 12', 'description': 'physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)'}, {'measure': 'automated visual field measurement', 'timeFrame': 'Month 15', 'description': 'physiological parameter : automated visual field corrected deviation of a visual field, measured according to a protocol STAT protocol 30 (Champ visuel Métrovision, Perenchies, France)'}, {'measure': 'Manual visual field measurement', 'timeFrame': 'Month 3', 'description': 'physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1'}, {'measure': 'Manual visual field measurement', 'timeFrame': 'Month 6', 'description': 'physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1'}, {'measure': 'Manual visual field measurement', 'timeFrame': 'Month 9', 'description': 'physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1'}, {'measure': 'Manual visual field measurement', 'timeFrame': 'Month 12', 'description': 'physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1'}, {'measure': 'Manual visual field measurement', 'timeFrame': 'Month 15', 'description': 'physiological parameter : manual visual field measured with isopters V / 4, III / 1 and II / 1'}, {'measure': 'National Eye Institute Visual Function Questionnaire 25', 'timeFrame': 'Month 12', 'description': 'Questionnaire'}, {'measure': 'Concentration of serum creatinine', 'timeFrame': 'Every three months until Month 15', 'description': 'Concentration of serum creatinine'}, {'measure': 'Concentration of high-density lipoprotein cholesterol', 'timeFrame': 'Every three months until Month 15', 'description': 'Blood test'}, {'measure': 'Concentration of very low-density lipoprotein cholesterol', 'timeFrame': 'Every three months until Month 15', 'description': 'Blood test'}, {'measure': 'Concentration of triglycerides', 'timeFrame': 'Every three months until Month 15', 'description': 'Blood test'}, {'measure': 'Rate of Partial thromboplastin time', 'timeFrame': 'Every three months until Month 15', 'description': 'Blood test'}, {'measure': 'Concentration of Aspartate aminotransférase', 'timeFrame': 'Every three months until Month 15', 'description': 'Blood test'}, {'measure': 'Concentration of Alanine aminotransférase', 'timeFrame': 'Every three months until Month 15', 'description': 'Blood test'}, {'measure': 'Concentration of lactate deshydrogenase', 'timeFrame': 'Every three months until Month 15', 'description': 'Blood test'}, {'measure': 'Concentration of creatine kinase', 'timeFrame': 'Every three months until Month 15', 'description': 'Blood test'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['leber herediatry optic neuropathy', 'befizal'], 'conditions': ['Safety Issues', 'Efficacy, Self']}, 'descriptionModule': {'briefSummary': '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', 'detailedDescription': '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'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* 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\n\nExclusion Criteria:\n\n* \\* Any optic neuropathy for which the diagnosis of LHON is not formally confirmed or genetically proven;\n\n * LHON that started for more than 5 years;\n * LHON associated with another primary mutation than 3460 or 11778\n * Children or adult patients under guardianship or deprived of liberty by administrative or judicial decision;\n * Women of childbearing age ; pregnant or lactating women;\n * Patients who do not have affiliation to a social protection scheme (national or private insurance / beneficiary or assignee);\n * Patient who did not give its written, informed and signed consent;\n * Allergy to fibrate, bezafibrate and / or BEFIZAL® 200mg (Arrow Generiques) or one of these constituents;\n * Photosensitivity reactions related to fibrates;\n * Patient already receiving treatment with fibrates or HMG Co-A reductase inhibitors or anticoagulants;\n * Hepatic insuffisiency or dysfunction with increased of transaminases (AST and ALT) over 3 times of the normal;\n * Renal insufficiency with serum creatinine\\> 15 mg / L (\\> 135 mg / dL) Biliary pathology'}, 'identificationModule': {'nctId': 'NCT04561466', 'acronym': 'Béfinohl', 'briefTitle': 'Trial of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy', 'organization': {'class': 'OTHER', 'fullName': 'Hôpital Necker-Enfants Malades'}, 'officialTitle': 'Study of Efficacy of Befizal® 200 mg for the Treatment of Leber Hereditary Optic Neuropathy', 'orgStudyIdInfo': {'id': 'beza16'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'treatment 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.\n\nBefizal® 200 mg will be tested for one year', 'interventionNames': ['Drug: Béfizal']}], 'interventions': [{'name': 'Béfizal', 'type': 'DRUG', 'description': '600 mf befizal a day for one year', 'armGroupLabels': ['treatment group']}]}, 'contactsLocationsModule': {'locations': [{'zip': '75015', 'city': 'Paris', 'country': 'France', 'facility': 'HEGP Hospital', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}], 'overallOfficials': [{'name': 'Dominique Bremond Gignac, MD PhD', 'role': 'STUDY_CHAIR', 'affiliation': 'Necker Hopsital'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED', 'description': 'Undecided'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Hôpital Necker-Enfants Malades', 'class': 'OTHER'}, 'collaborators': [{'name': 'European Georges Pompidou Hospital', 'class': 'OTHER'}, {'name': 'CLAIROP', 'class': 'UNKNOWN'}], 'responsibleParty': {'type': 'PRINCIPAL_INVESTIGATOR', 'investigatorTitle': 'Principal investigator', 'investigatorFullName': 'Christophe Orssaud', 'investigatorAffiliation': 'Hôpital Necker-Enfants Malades'}}}}