Ignite Creation Date:
2025-12-26 @ 10:44 PM
Ignite Modification Date:
2025-12-26 @ 10:44 PM
Study NCT ID:
NCT02660112
Status:
COMPLETED
Last Update Posted:
2019-12-05
First Post:
2016-01-18
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
(+) Epicatechin to Treat Friedreich's Ataxia
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D005621', 'term': 'Friedreich Ataxia'}], 'ancestors': [{'id': 'D013132', 'term': 'Spinocerebellar Degenerations'}, {'id': 'D002526', 'term': 'Cerebellar Diseases'}, {'id': 'D001927', 'term': 'Brain Diseases'}, {'id': 'D002493', 'term': 'Central Nervous System Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D013118', 'term': 'Spinal Cord Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D028361', 'term': 'Mitochondrial Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D002392', 'term': 'Catechin'}], 'ancestors': [{'id': 'D002839', 'term': 'Chromans'}, {'id': 'D001578', 'term': 'Benzopyrans'}, {'id': 'D011714', 'term': 'Pyrans'}, {'id': 'D006573', 'term': 'Heterocyclic Compounds, 1-Ring'}, {'id': 'D006571', 'term': 'Heterocyclic Compounds'}, {'id': 'D005419', 'term': 'Flavonoids'}, {'id': 'D002867', 'term': 'Chromones'}, {'id': 'D006574', 'term': 'Heterocyclic Compounds, 2-Ring'}, {'id': 'D000072471', 'term': 'Heterocyclic Compounds, Fused-Ring'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'Gavrilova.Ralitza@mayo.edu', 'phone': '507-293-1283', 'title': 'Ralitza H. Gavrilova, M.D.', 'organization': 'Mayo Clinic'}, 'certainAgreement': {'piSponsorEmployee': True}}, 'adverseEventsModule': {'timeFrame': 'Adverse Events were collected over a 24 week time period', 'eventGroups': [{'id': 'EG000', 'title': '(+)-Epicatechin', 'description': 'Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks\n\n(+)-Epicatechin: 25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)', 'otherNumAtRisk': 10, 'deathsNumAtRisk': 10, 'otherNumAffected': 3, 'seriousNumAtRisk': 10, 'deathsNumAffected': 0, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Transient worsening of migraine', 'stats': [{'groupId': 'EG000', 'numAtRisk': 10, 'numAffected': 3}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT'}], 'frequencyThreshold': '0'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score', 'denoms': [{'units': 'Participants', 'counts': [{'value': '9', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': '(+)-Epicatechin', 'description': 'Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks\n\n(+)-Epicatechin: 25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)'}], 'classes': [{'categories': [{'measurements': [{'value': '55.0', 'spread': '18.72', 'groupId': 'OG000'}]}]}], 'analyses': [{'pValue': '0.336', 'groupIds': ['OG000'], 'statisticalMethod': 'Wilcoxon (Mann-Whitney)', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, 24 weeks', 'description': 'The Friedreich Ataxia Rating Scale (FARS) is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate (assesses speech speed by repetitions of pronouncing "PaTa" ) and low-contrast letter acuity.\n\nFARS is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate and low-contrast letter acuity. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.', 'unitOfMeasure': 'score on a scale', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED', 'populationDescription': 'One subject did not complete week 24 questionnaire'}, {'type': 'PRIMARY', 'title': 'Change in Ventricular Hypertrophy as Shown on Cardiac MRI', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'OG000'}]}], 'groups': [{'id': 'OG000', 'title': '(+)-Epicatechin', 'description': 'Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks\n\n(+)-Epicatechin: 25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)'}], 'classes': [{'categories': [{'measurements': [{'value': '55.9', 'spread': '9.49', 'groupId': 'OG000'}]}]}], 'paramType': 'MEAN', 'timeFrame': 'Baseline, 24 weeks', 'description': 'Left ventricular mass and left ventricular (LV) mass indexed to body surface area estimated by Left Ventricle (LV)cavity dimension and wall thickness at end-diastole. Normal values of LV mass indexed to body surface area are found to be 49-115 gL/m2 in men and 43-95 g/m2 in women.', 'unitOfMeasure': 'mL/m2', 'dispersionType': 'Standard Deviation', 'reportingStatus': 'POSTED'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': '(+)-Epicatechin', 'description': 'Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks\n\n(+)-Epicatechin: 25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '10'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'groups': [{'id': 'BG000', 'title': '(+)-Epicatechin', 'description': 'Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks\n\n(+)-Epicatechin: 25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '14.5', 'groupId': 'BG000', 'lowerLimit': '10', 'upperLimit': '50'}]}]}], 'paramType': 'MEDIAN', 'unitOfMeasure': 'years', 'dispersionType': 'INTER_QUARTILE_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'categories': [{'title': 'Female', 'measurements': [{'value': '4', 'groupId': 'BG000'}]}, {'title': 'Male', 'measurements': [{'value': '6', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race and Ethnicity Not Collected', 'classes': [{'denoms': [{'units': 'Participants', 'counts': [{'value': '0', 'groupId': 'BG000'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants', 'populationDescription': 'Race and Ethnicity were not collected from any participant.'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'United States', 'denoms': [{'units': 'Participants', 'counts': [{'value': '10', 'groupId': 'BG000'}]}], 'categories': [{'measurements': [{'value': '10', 'groupId': 'BG000'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2017-01-27', 'size': 1556390, 'label': 'Study Protocol and Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'Prot_SAP_000.pdf', 'typeAbbrev': 'Prot_SAP', 'uploadDate': '2019-11-14T09:59', 'hasProtocol': True}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 10}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2016-09'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2019-02', 'completionDateStruct': {'date': '2018-12-30', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2019-11-18', 'studyFirstSubmitDate': '2016-01-18', 'resultsFirstSubmitDate': '2019-11-18', 'studyFirstSubmitQcDate': '2016-01-18', 'lastUpdatePostDateStruct': {'date': '2019-12-05', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2019-11-18', 'studyFirstPostDateStruct': {'date': '2016-01-21', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2019-12-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2018-12-30', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Friedreich Ataxia Rating Scale (FARS) Composite Score', 'timeFrame': 'Baseline, 24 weeks', 'description': 'The Friedreich Ataxia Rating Scale (FARS) is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate (assesses speech speed by repetitions of pronouncing "PaTa" ) and low-contrast letter acuity.\n\nFARS is made up of a measure of ataxia, an activities of daily living subscale and a neurological subscale. This scale also includes the 8m walk at maximum speed (8MW), the 9-hole peg test (9HPT), PATA rate and low-contrast letter acuity. The scores from the three subscales are added to generate a total score ranging from 0 to 159, with a higher score indicating a greater level of disability.'}, {'measure': 'Change in Ventricular Hypertrophy as Shown on Cardiac MRI', 'timeFrame': 'Baseline, 24 weeks', 'description': 'Left ventricular mass and left ventricular (LV) mass indexed to body surface area estimated by Left Ventricle (LV)cavity dimension and wall thickness at end-diastole. Normal values of LV mass indexed to body surface area are found to be 49-115 gL/m2 in men and 43-95 g/m2 in women.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'conditions': ["Friedreich's Ataxia"]}, 'referencesModule': {'references': [{'pmid': '32677106', 'type': 'DERIVED', 'citation': "Qureshi MY, Patterson MC, Clark V, Johnson JN, Moutvic MA, Driscoll SW, Kemppainen JL, Huston J 3rd, Anderson JR, Badley AD, Tebben PJ, Wackel P, Oglesbee D, Glockner J, Schreiner G, Dugar S, Touchette JC, Gavrilova RH. Safety and efficacy of (+)-epicatechin in subjects with Friedreich's ataxia: A phase II, open-label, prospective study. J Inherit Metab Dis. 2021 Mar;44(2):502-514. doi: 10.1002/jimd.12285. Epub 2020 Aug 31."}]}, 'descriptionModule': {'briefSummary': "This 24-week study will test the safety and effectiveness of synthetically produced (+) Epicatechin in treating patients who have Friedreich's Ataxia, a neurological disorder."}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '50 Years', 'minimumAge': '10 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* Confirmed diagnosis of Friedreich's Ataxia (FA) by Frataxin genetic testing and/or Frataxin enzyme analysis\n* Between age 10 and 50 years of age, inclusive\n* Body weight of 25 kilograms or higher\n* Minimum of one affected organ (cardiac or neurological) system, as evidenced by clinical signs/symptoms\n* Disease duration ≤7 years, based on onset date of FA symptoms\n* Has no known contraindication to gadolinium contrast such as severe allergy or Glomerular Filtration Rate \\<30 ml/min/m\\^2.\n* Has no known contraindication to non-contrast Magnetic Resonance Imaging (MRI) evaluation such as pacemaker or magnetically active metal fragments.\n* Women of childbearing age must:\n\n * Have a negative pregnancy human chorionic gonadotropin test prior to receiving study drug.\n * Agree to use contraception for the duration of the study drug dosing, plus 1 month after completion of the study.\n\nExclusion Criteria:\n\n* Advanced cardiac failure, New York Heart Association (NYHA) Classification Scale-Class IV (advanced stage heart failure)\n* Clinically significant comorbidities that may also lead to cardiomyopathy, for example long standing hypertension, familial cardiomyopathy.\n* Clinically significant comorbidities that would, in the opinion of the investigators, compromise the interpretation of test results.\n* Pregnant, breast-feeding or planning to become pregnant during study timeframe.\n* Patients with contraindications to regadenoson, i. e. second- or third-degree atrioventricular (AV) block or sinus node dysfunction. Has received an investigational drug within thirty (30) days of baseline visit.\n* Thrombocytopenia (\\<125 x 10\\^9/Liter) or prolonged Prothrombin Time/Partial Thromboplastin Time (PT/PTT) at baseline.\n* Clinically significant hypotension (systolic blood pressure \\<90) due to heart failure or other conditions."}, 'identificationModule': {'nctId': 'NCT02660112', 'briefTitle': "(+) Epicatechin to Treat Friedreich's Ataxia", 'organization': {'class': 'OTHER', 'fullName': 'Mayo Clinic'}, 'officialTitle': "A Phase II, Open Label Prospective Single Center Drug Study Evaluating the Safety and Efficacy of (+)-Epicatechin in Subjects With Friedreich's Ataxia", 'orgStudyIdInfo': {'id': '15-006845'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '(+)-Epicatechin', 'description': 'Total daily dose 75mg (+)-Epicatechin; 25mg cap three times per day by mouth for 24 weeks', 'interventionNames': ['Drug: (+)-Epicatechin']}], 'interventions': [{'name': '(+)-Epicatechin', 'type': 'DRUG', 'description': '25mg (+)-Epicatechin capsules. Starting dose 75mg total daily dose (one 25mg cap taken three times per day). Dose escalation at 12 weeks for non-responders to 150mg total daily dose (two 25mg caps taken three times per day)', 'armGroupLabels': ['(+)-Epicatechin']}]}, 'contactsLocationsModule': {'locations': [{'zip': '55905', 'city': 'Rochester', 'state': 'Minnesota', 'country': 'United States', 'facility': 'Mayo Clinic', 'geoPoint': {'lat': 44.02163, 'lon': -92.4699}}], 'overallOfficials': [{'name': 'Ralitza H Gavrilova, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Mayo Clinic'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Ralitza Gavrilova', 'class': 'OTHER'}, 'collaborators': [{'name': 'Cardero Therapeutics, Inc.', 'class': 'INDUSTRY'}], 'responsibleParty': {'type': 'SPONSOR_INVESTIGATOR', 'investigatorTitle': 'MD, Sponsor-Investigator', 'investigatorFullName': 'Ralitza Gavrilova', 'investigatorAffiliation': 'Mayo Clinic'}}}}