Ignite Creation Date:
2025-12-24 @ 10:01 PM
Ignite Modification Date:
2026-01-04 @ 12:09 AM
Study NCT ID:
NCT01995032
Status:
COMPLETED
Last Update Posted:
2018-04-19
First Post:
2013-11-20
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
L-citrulline and Metformin in Duchenne's Muscular Dystrophy
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D020388', 'term': 'Muscular Dystrophy, Duchenne'}], 'ancestors': [{'id': 'D009136', 'term': 'Muscular Dystrophies'}, {'id': 'D020966', 'term': 'Muscular Disorders, Atrophic'}, {'id': 'D009135', 'term': 'Muscular Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D040181', 'term': 'Genetic Diseases, X-Linked'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'D008687', 'term': 'Metformin'}], 'ancestors': [{'id': 'D001645', 'term': 'Biguanides'}, {'id': 'D006146', 'term': 'Guanidines'}, {'id': 'D000578', 'term': 'Amidines'}, {'id': 'D009930', 'term': 'Organic Chemicals'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 47}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2013-10'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2018-04', 'completionDateStruct': {'date': '2016-03', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2018-04-17', 'studyFirstSubmitDate': '2013-11-20', 'studyFirstSubmitQcDate': '2013-11-20', 'lastUpdatePostDateStruct': {'date': '2018-04-19', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2013-11-26', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2015-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Mean change of motor function measure (MFM) D1 subscore (assessing standing and transfers)', 'timeFrame': 'baseline to week 26'}], 'secondaryOutcomes': [{'measure': 'Mean change of MFM total score, the D2, and D3 MFM subscores', 'timeFrame': 'baseline to week 26'}, {'measure': 'Mean change of six minute walking distance (6MWD)', 'timeFrame': 'baseline to week 26'}, {'measure': 'Change of quantitative muscle MRI (Magnetic Resonance Imaging) including muscle fat content (MFC) and T2 times of thigh muscles', 'timeFrame': 'baseline to week 26'}, {'measure': 'Change in the plasma/urine concentration for markers of muscle necrosis, oxidative stress, nitrosative stress, and change of microRNA (miRNA)', 'timeFrame': 'baseline to week 26'}, {'measure': 'Mean change of quantitative muscle force (QMT) of knee extension and elbow flexion using hand held dynamometry (HHD)', 'timeFrame': 'baseline to week 26'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ["Duchenne's muscular dystrophy", 'DMD'], 'conditions': ["Duchenne's Muscular Dystrophy (DMD)"]}, 'referencesModule': {'references': [{'pmid': '31664444', 'type': 'DERIVED', 'citation': 'Hafner P, Bonati U, Klein A, Rubino D, Gocheva V, Schmidt S, Schroeder J, Bernert G, Laugel V, Steinlin M, Capone A, Gloor M, Bieri O, Hemkens LG, Speich B, Zumbrunn T, Gueven N, Fischer D. Effect of Combination l-Citrulline and Metformin Treatment on Motor Function in Patients With Duchenne Muscular Dystrophy: A Randomized Clinical Trial. JAMA Netw Open. 2019 Oct 2;2(10):e1914171. doi: 10.1001/jamanetworkopen.2019.14171.'}, {'pmid': '27488051', 'type': 'DERIVED', 'citation': 'Hafner P, Bonati U, Rubino D, Gocheva V, Zumbrunn T, Gueven N, Fischer D. Treatment with L-citrulline and metformin in Duchenne muscular dystrophy: study protocol for a single-centre, randomised, placebo-controlled trial. Trials. 2016 Aug 3;17(1):389. doi: 10.1186/s13063-016-1503-1.'}]}, 'descriptionModule': {'briefSummary': "The purpose of the study is to show that the intake of L-citrulline and metformin improves muscle function and delay of progression in patients with Duchenne's muscular dystrophy.", 'detailedDescription': 'This is a single center, randomized, double-blind, placebo controlled study. The study medication consists of L-citrulline and metformin, respectively placebo given orally; 2.5 g L-citrulline or placebo will be given 3 times daily, metformin containing 250 mg or placebo will be administered 3 times daily. The duration of the study is 26 weeks and comprehends one screening and three study visits.\n\nAmendment 1: Amended eligibility criteria'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '10 Years', 'minimumAge': '78 Months', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Molecular diagnosis of DMD\n* Patients 6.5 - 10 years of age at time of screening\n* Ambulant\n* Ability to walk 150 m in the 6 min walking distance (6MWT)\n* D1 subdomain of the MFM scale \\>40%\n* stable treatment with steroids for \\>6 months or steroid naïve patients\n\nExclusion Criteria:\n\n* Previous (3 months or less) or concomitant participation in another therapeutic trial\n* Use of L-citrulline, L-arginine or metformin within the last 3 months\n* Known individual hypersensitivity to L-citrulline or metformin\n* known or suspected malignancy\n* Other chronic disease or clinical relevant limitation of renal, liver, heart function according to discretion of investigator\n* start of cortisone treatment or change in dosage \\<6 months prior to screening'}, 'identificationModule': {'nctId': 'NCT01995032', 'briefTitle': "L-citrulline and Metformin in Duchenne's Muscular Dystrophy", 'organization': {'class': 'OTHER', 'fullName': 'University Hospital, Basel, Switzerland'}, 'officialTitle': '"A Double Blind Randomised Placebo Controlled Efficacy and Safety Study of L-citrulline and Metformin in Ambulant Children Aged Between 7 and 10 Years With Duchenne\'s Muscular Dystrophy"', 'orgStudyIdInfo': {'id': 'DMD02'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': '750 mg metformin and 7.5 g L-citrulline daily p.o.', 'description': '7.5 g L-citrulline p.o. and 750 mg metformin daily p.o. (3x 2.5 g, respectively 3x 250 mg) for 26 weeks', 'interventionNames': ['Drug: 750 mg metformin and 7.5 g L-citrulline daily p.o.']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'metformin placebo and L-citrulline placebo 3 times daily p.o. for 26 weeks', 'interventionNames': ['Drug: Placebo']}], 'interventions': [{'name': '750 mg metformin and 7.5 g L-citrulline daily p.o.', 'type': 'DRUG', 'armGroupLabels': ['750 mg metformin and 7.5 g L-citrulline daily p.o.']}, {'name': 'Placebo', 'type': 'DRUG', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '4031', 'city': 'Basel', 'state': 'Canton of Basel-City', 'country': 'Switzerland', 'facility': "University Children's Hospital", 'geoPoint': {'lat': 47.55839, 'lon': 7.57327}}], 'overallOfficials': [{'name': 'Dirk Fischer, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': "University Children's Hospital Basel"}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University Hospital, Basel, Switzerland', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}