Ignite Creation Date:
2025-12-24 @ 6:38 PM
Ignite Modification Date:
2026-02-27 @ 10:39 AM
Study NCT ID:
NCT01401257
Status:
COMPLETED
Last Update Posted:
2017-11-22
First Post:
2011-07-20
Is Gene Therapy:
True
Has Adverse Events:
True
Brief Title:
Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002607', 'term': 'Charcot-Marie-Tooth Disease'}, {'id': 'C536965', 'term': 'Tomaculous neuropathy'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D015417', 'term': 'Hereditary Sensory and Motor Neuropathy'}], 'ancestors': [{'id': 'D009421', 'term': 'Nervous System Malformations'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D011115', 'term': 'Polyneuropathies'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'shahram.attarian@ap-hm.fr', 'phone': '04 91 38 65', 'title': 'Pr Shahram ATTARIAN', 'phoneExt': '79', 'organization': 'Hôpital La Timone, Marseille'}, 'certainAgreement': {'otherDetails': 'No individual publication (paper, abstract, poster, oral presentation) is allowed until results of the complete study have been published.\n\nThe Investigator agrees that any proposed publication, presentation or use of results arising from the Investigation will be submitted to Pharnext for review and written agreement prior to submission.', 'restrictionType': 'OTHER', 'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': '13 months', 'eventGroups': [{'id': 'EG000', 'title': 'PXT3003 Low Dose', 'description': 'Oral Liquid formulation, 1/100, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment', 'otherNumAtRisk': 21, 'otherNumAffected': 12, 'seriousNumAtRisk': 21, 'seriousNumAffected': 2}, {'id': 'EG001', 'title': 'PXT3003 Intermediate Dose', 'description': 'Oral Liquid formulation, 1/50, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment', 'otherNumAtRisk': 21, 'otherNumAffected': 10, 'seriousNumAtRisk': 21, 'seriousNumAffected': 0}, {'id': 'EG002', 'title': 'PXT3003 High Dose', 'description': 'Oral Liquid formulation, 1/10, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment', 'otherNumAtRisk': 19, 'otherNumAffected': 13, 'seriousNumAtRisk': 19, 'seriousNumAffected': 0}, {'id': 'EG003', 'title': 'Placebo', 'description': 'Oral Liquid formulation, bid, 12 months\n\nPlacebo: Liquid,5 ml, twice a day, 12-month treatment', 'otherNumAtRisk': 19, 'otherNumAffected': 11, 'seriousNumAtRisk': 19, 'seriousNumAffected': 0}], 'otherEvents': [{'term': 'Infections and infestations', 'stats': [{'groupId': 'EG000', 'numAtRisk': 21, 'numAffected': 11}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 10}, {'groupId': 'EG002', 'numAtRisk': 19, 'numAffected': 11}, {'groupId': 'EG003', 'numAtRisk': 19, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.1'}, {'term': 'Musculoskeletal and connective tissue disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 21, 'numAffected': 12}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 8}, {'groupId': 'EG002', 'numAtRisk': 19, 'numAffected': 13}, {'groupId': 'EG003', 'numAtRisk': 19, 'numAffected': 7}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.1'}, {'term': 'Nervous system disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 21, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 8}, {'groupId': 'EG002', 'numAtRisk': 19, 'numAffected': 11}, {'groupId': 'EG003', 'numAtRisk': 19, 'numAffected': 9}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.1'}, {'term': 'Gastrointestinal disorders', 'stats': [{'groupId': 'EG000', 'numAtRisk': 21, 'numAffected': 7}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 5}, {'groupId': 'EG002', 'numAtRisk': 19, 'numAffected': 10}, {'groupId': 'EG003', 'numAtRisk': 19, 'numAffected': 11}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.1'}], 'seriousEvents': [{'term': 'osteoarthritis', 'notes': 'Unrelated to study medication', 'stats': [{'groupId': 'EG000', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 19, 'numAffected': 0}], 'organSystem': 'Musculoskeletal and connective tissue disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.1'}, {'term': 'Hemorrhoids', 'notes': 'Unrelated to study medication.', 'stats': [{'groupId': 'EG000', 'numAtRisk': 21, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 21, 'numAffected': 0}, {'groupId': 'EG002', 'numAtRisk': 19, 'numAffected': 0}, {'groupId': 'EG003', 'numAtRisk': 19, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 13.1'}], 'frequencyThreshold': '2'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Safety and Tolerability of PXT3003', 'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}, {'value': '19', 'groupId': 'OG002'}, {'value': '19', 'groupId': 'OG003'}]}], 'groups': [{'id': 'OG000', 'title': 'PXT3003 Low Dose', 'description': 'Oral Liquid formulation, 1/100, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'OG001', 'title': 'PXT3003 Intermediate Dose', 'description': 'Oral Liquid formulation, 1/50, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'OG002', 'title': 'PXT3003 High Dose', 'description': 'Oral Liquid formulation, 1/10, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'OG003', 'title': 'Placebo', 'description': 'Oral Liquid formulation, bid, 12 months\n\nPlacebo: Liquid,5 ml, twice a day, 12-month treatment'}], 'classes': [{'categories': [{'measurements': [{'value': '21', 'groupId': 'OG000'}, {'value': '21', 'groupId': 'OG001'}, {'value': '19', 'groupId': 'OG002'}, {'value': '19', 'groupId': 'OG003'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'timeFrame': 'Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up', 'description': 'The Primary Objective is to assess the clinical and laboratory safety and tolerability of three doses of PXT3003 administered orally for 12 months to CMT1A patients versus placebo.\n\nNumber of participants with adverse events in each arm.', 'unitOfMeasure': 'Participants', 'reportingStatus': 'POSTED'}, {'type': 'SECONDARY', 'title': 'To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests', 'timeFrame': 'Screening, randomization, 3-, 6-, 9- and 12-months treatment', 'description': 'Efficacy scores and functional tests will be assessed CMTNS/CMTES:ONLS, VAS, fatigue, pain, six minute walk test (6MWT), nine-hole peg test, quantified muscular testing (QMT; hand grip and foot dorsiflexion), CGI.\n\nFor each test or score, change from baseline after 3-,6-, 9- and 12-months of treatment.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy', 'timeFrame': 'Randomization and 12-month treatment', 'description': 'A cutaneous biopsy (consisting in 2 small punch biopsies) will be performed to assess PMP22 mRNA expression and intra-epidermal axon density.\n\nChange from baseline after 12-month of treatment.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters', 'timeFrame': 'Screening, randomization, 3-, 6-, 9- and 12-month treatment', 'description': 'Electrophysiological examination will be performed to assess sensory and motor responses of the median and ulnar nerves (non-dominant side) including: NCV, compound muscle action potential (CMAP) and SNAP.\n\nChange from baseline after 3-,6-, 9- and 12-months of treatment.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers', 'timeFrame': 'Randomization and 3-month treatment', 'description': 'Dosages of biochemical biomarkers in plasma.\n\nChange from baseline after 3-month of treatment.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'To Assess the Plasma Concentrations of PXT3003', 'timeFrame': 'Randomization, 1-, 6- and 12-month treatment', 'description': 'PXT3003 plasmatic concentrations after one administration (randomization) and after 1-,6-and 12-months of treatment.', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'PXT3003 Low Dose', 'description': 'Oral Liquid formulation, 1/100, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'FG001', 'title': 'PXT3003 Intermediate Dose', 'description': 'Oral Liquid formulation, 1/50, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'FG002', 'title': 'PXT3003 High Dose', 'description': 'Oral Liquid formulation, 1/10, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'FG003', 'title': 'Placebo', 'description': 'Oral Liquid formulation, bid, 12 months\n\nPlacebo: Liquid,5 ml, twice a day, 12-month treatment'}], 'periods': [{'title': 'Overall Study', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}, {'groupId': 'FG001', 'numSubjects': '21'}, {'groupId': 'FG002', 'numSubjects': '19'}, {'groupId': 'FG003', 'numSubjects': '19'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '21'}, {'groupId': 'FG001', 'numSubjects': '19'}, {'groupId': 'FG002', 'numSubjects': '16'}, {'groupId': 'FG003', 'numSubjects': '17'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}, {'groupId': 'FG002', 'numSubjects': '3'}, {'groupId': 'FG003', 'numSubjects': '2'}]}]}]}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '21', 'groupId': 'BG000'}, {'value': '21', 'groupId': 'BG001'}, {'value': '19', 'groupId': 'BG002'}, {'value': '19', 'groupId': 'BG003'}, {'value': '80', 'groupId': 'BG004'}]}], 'groups': [{'id': 'BG000', 'title': 'PXT3003 Low Dose', 'description': 'Oral Liquid formulation, 1/100, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'BG001', 'title': 'PXT3003 Intermediate Dose', 'description': 'Oral Liquid formulation, 1/50, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'BG002', 'title': 'PXT3003 High Dose', 'description': 'Oral Liquid formulation, 1/10, bid, 12 months\n\nPXT3003: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'BG003', 'title': 'Placebo', 'description': 'Oral Liquid formulation, bid, 12 months\n\nPlacebo: Liquid,5 ml, twice a day, 12-month treatment'}, {'id': 'BG004', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Categorical', 'classes': [{'categories': [{'title': '<=18 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}, {'title': 'Between 18 and 65 years', 'measurements': [{'value': '21', 'groupId': 'BG000'}, {'value': '21', 'groupId': 'BG001'}, {'value': '19', 'groupId': 'BG002'}, {'value': '19', 'groupId': 'BG003'}, {'value': '80', 'groupId': 'BG004'}]}, {'title': '>=65 years', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '0', 'groupId': 'BG001'}, {'value': '0', 'groupId': 'BG002'}, {'value': '0', 'groupId': 'BG003'}, {'value': '0', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '47.9', 'groupId': 'BG000', 'lowerLimit': '18', 'upperLimit': '63'}, {'value': '44.3', 'groupId': 'BG001', 'lowerLimit': '23', 'upperLimit': '64'}, {'value': '44.6', 'groupId': 'BG002', 'lowerLimit': '24', 'upperLimit': '63'}, {'value': '43.2', 'groupId': 'BG003', 'lowerLimit': '21', 'upperLimit': '65'}, {'value': '45.1', 'groupId': 'BG004', 'lowerLimit': '18', 'upperLimit': '65'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'FULL_RANGE'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '14', 'groupId': 'BG000'}, {'value': '13', 'groupId': 'BG001'}, {'value': '10', 'groupId': 'BG002'}, {'value': '11', 'groupId': 'BG003'}, {'value': '48', 'groupId': 'BG004'}]}, {'title': 'Male', 'measurements': [{'value': '7', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}, {'value': '8', 'groupId': 'BG003'}, {'value': '32', 'groupId': 'BG004'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Region of Enrollment', 'classes': [{'title': 'France', 'categories': [{'measurements': [{'value': '21', 'groupId': 'BG000'}, {'value': '21', 'groupId': 'BG001'}, {'value': '19', 'groupId': 'BG002'}, {'value': '19', 'groupId': 'BG003'}, {'value': '80', 'groupId': 'BG004'}]}]}], 'paramType': 'NUMBER', 'unitOfMeasure': 'participants'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'QUADRUPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR', 'OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 80}}, 'statusModule': {'overallStatus': 'COMPLETED', 'startDateStruct': {'date': '2010-12'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2017-10', 'completionDateStruct': {'date': '2012-12', 'type': 'ACTUAL'}, 'lastUpdateSubmitDate': '2017-10-19', 'studyFirstSubmitDate': '2011-07-20', 'resultsFirstSubmitDate': '2016-12-23', 'studyFirstSubmitQcDate': '2011-07-22', 'lastUpdatePostDateStruct': {'date': '2017-11-22', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2016-12-23', 'studyFirstPostDateStruct': {'date': '2011-07-25', 'type': 'ESTIMATED'}, 'resultsFirstPostDateStruct': {'date': '2017-02-16', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2011-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Safety and Tolerability of PXT3003', 'timeFrame': 'Screening, randomization, 1-, 3-, 6-, 9-, 12-month treatment and 1-month follow-up', 'description': 'The Primary Objective is to assess the clinical and laboratory safety and tolerability of three doses of PXT3003 administered orally for 12 months to CMT1A patients versus placebo.\n\nNumber of participants with adverse events in each arm.'}], 'secondaryOutcomes': [{'measure': 'To Obtain Preliminary Data on the Efficacy of PXT3003 on Clinical Scores and Functional Tests', 'timeFrame': 'Screening, randomization, 3-, 6-, 9- and 12-months treatment', 'description': 'Efficacy scores and functional tests will be assessed CMTNS/CMTES:ONLS, VAS, fatigue, pain, six minute walk test (6MWT), nine-hole peg test, quantified muscular testing (QMT; hand grip and foot dorsiflexion), CGI.\n\nFor each test or score, change from baseline after 3-,6-, 9- and 12-months of treatment.'}, {'measure': 'To Assess the Pharmacodynamic Effect of PXT3003 on PMP22 mRNA Levels and Intra-epidermal Axon Density in Cutaneous Biopsy', 'timeFrame': 'Randomization and 12-month treatment', 'description': 'A cutaneous biopsy (consisting in 2 small punch biopsies) will be performed to assess PMP22 mRNA expression and intra-epidermal axon density.\n\nChange from baseline after 12-month of treatment.'}, {'measure': 'To Assess the Pharmacodynamic Effect of PXT3003 on Selected Neurophysiological Parameters', 'timeFrame': 'Screening, randomization, 3-, 6-, 9- and 12-month treatment', 'description': 'Electrophysiological examination will be performed to assess sensory and motor responses of the median and ulnar nerves (non-dominant side) including: NCV, compound muscle action potential (CMAP) and SNAP.\n\nChange from baseline after 3-,6-, 9- and 12-months of treatment.'}, {'measure': 'To Assess the Pharmacodynamic Effect of PXT3003 on a Series of Biochemical Biomarkers', 'timeFrame': 'Randomization and 3-month treatment', 'description': 'Dosages of biochemical biomarkers in plasma.\n\nChange from baseline after 3-month of treatment.'}, {'measure': 'To Assess the Plasma Concentrations of PXT3003', 'timeFrame': 'Randomization, 1-, 6- and 12-month treatment', 'description': 'PXT3003 plasmatic concentrations after one administration (randomization) and after 1-,6-and 12-months of treatment.'}]}, 'oversightModule': {'oversightHasDmc': True}, 'conditionsModule': {'keywords': ['PXT3003', 'Charcot-Marie-Tooth Disease', 'Hereditary Motor and Sensory Neuropathies'], 'conditions': ['Charcot-Marie-Tooth Disease', 'Hereditary Neuropathy With Liability to Pressure Palsies', 'Genetic Disorders']}, 'referencesModule': {'references': [{'pmid': '25519680', 'type': 'DERIVED', 'citation': 'Attarian S, Vallat JM, Magy L, Funalot B, Gonnaud PM, Lacour A, Pereon Y, Dubourg O, Pouget J, Micallef J, Franques J, Lefebvre MN, Ghorab K, Al-Moussawi M, Tiffreau V, Preudhomme M, Magot A, Leclair-Visonneau L, Stojkovic T, Bossi L, Lehert P, Gilbert W, Bertrand V, Mandel J, Milet A, Hajj R, Boudiaf L, Scart-Gres C, Nabirotchkin S, Guedj M, Chumakov I, Cohen D. An exploratory randomised double-blind and placebo-controlled phase 2 study of a combination of baclofen, naltrexone and sorbitol (PXT3003) in patients with Charcot-Marie-Tooth disease type 1A. Orphanet J Rare Dis. 2014 Dec 18;9:199. doi: 10.1186/s13023-014-0199-0.'}]}, 'descriptionModule': {'briefSummary': 'The present trial is a randomized, placebo-controlled study evaluating 3 different doses of PXT3003 in patients with CMT1A disease.', 'detailedDescription': 'In addition to the safety and tolerability of the treatment, clinical, electrophysiological and biological endpoints (PMP22 mRNA, skin biopsy histology and plasma biomarkers) will be assessed. Standard laboratory tests and drug plasma concentrations will also be measured. Because of the slow progression of the disease and the nature of the observed symptoms, a minimum duration of 12 months of treatment is required in order to observe a potential improvement in any of the efficacy parameters.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '65 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* DNA proven CMT1A\n* Muscle weakness in at least foot dorsiflexion (clinical assessment)\n* Age between 18 and 65 years\n* Male or non pregnant, non breastfeeding female\n* CMT neuropathy score at screening ≤ 20\n* Agrees to perform electrorophysiological studies and two cutaneous biopsies for determination of PMP22 expression and histology\n* Providing signed written informed consent to participate in the study and willing and able to comply with all study procedures and scheduled visits\n\nExclusion Criteria:\n\n* Patients with another neurological disease\n* Patients using unauthorized concomitant treatments, ascorbic acid, opioids, levothyroxine and potentially neurotoxic drugs. Patients who can/agree to stop these medications 4 weeks before randomization can be included\n* Patients who have participated in another trial of investigational drug within the past 30 days\n* Concomitant major systemic disease\n* Clinically significant history of unstable medical illness over the last 30 days (unstable angina…)\n* History of significant hematologic, kidney, liver disease, or insulin-dependent diabetes\n* Clinically significant abnormalities on the prestudy laboratory evaluation, physical evaluation, electrocardiogram (ECG)\n* ASAT/ALAT levels above the upper limit of normal (ULN). However, patients with an isolated elevation of either ASAT or ALAT (\\<1.5 ULN) can be included at investigators" discretion if the remaining liver function tests are normal and if ASAT or ALAT value is stable at 2 distinct evaluations in the month prior to inclusion\n* Serum creatinine levels above the upper limit of normal\n* Limited mental capacity or psychiatric disease rendering the subject unable to provide written informed consent or comply with evaluation procedures\n* History of recent alcohol or drug abuse or non-adherence with treatment or other experimental protocols\n* Female of childbearing potential (apart of patient using adequate contraceptive measures), pregnant or breast feeding\n* Suspected inability to complete the study follow-up (foreign workers, transient visitors, tourists or any others for whom follow-up evaluation is not assured)\n* Limb surgery in the six months before randomization or planned before completion of the trial\n* Known hypersensitivity to any of the individual components of PXT3003\n* Porphyria'}, 'identificationModule': {'nctId': 'NCT01401257', 'briefTitle': 'Phase II, Randomized, Placebo-controlled Trial in Patients With Charcot-marie-tooth Disease Type 1A', 'organization': {'class': 'OTHER', 'fullName': 'Pharnext S.C.A.'}, 'officialTitle': 'A Phase II, Randomized, Placebo-controlled Trial of the Safety, Efficacy, Pharmacodynamics and Pharmacokinetics of PXT3003 in Patients With Charcot-Marie-Tooth Disease Type 1A.', 'orgStudyIdInfo': {'id': 'CLN-PXT3003-01'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'PXT3003 Low dose', 'description': 'Oral Liquid formulation, 1/100, bid, 12 months', 'interventionNames': ['Drug: PXT3003 Low dose']}, {'type': 'EXPERIMENTAL', 'label': 'PXT3003 Intermediate dose', 'description': 'Oral Liquid formulation, 1/50, bid, 12 months', 'interventionNames': ['Drug: PXT3003 Intermediate Dose']}, {'type': 'EXPERIMENTAL', 'label': 'PXT3003 High dose', 'description': 'Oral Liquid formulation, 1/10, bid, 12 months', 'interventionNames': ['Drug: PXT3003 High Dose']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Oral Liquid formulation, bid, 12 months', 'interventionNames': ['Other: Placebo']}], 'interventions': [{'name': 'PXT3003 Low dose', 'type': 'DRUG', 'otherNames': ['Pleocompound PXT3003'], 'description': 'Liquid,5 ml, twice a day, 12-month treatment', 'armGroupLabels': ['PXT3003 Low dose']}, {'name': 'PXT3003 Intermediate Dose', 'type': 'DRUG', 'otherNames': ['Pleocompound PXT3003'], 'description': 'Liquid,5 ml, twice a day, 12-month treatment', 'armGroupLabels': ['PXT3003 Intermediate dose']}, {'name': 'PXT3003 High Dose', 'type': 'DRUG', 'otherNames': ['Pleocompound PXT3003'], 'description': 'Liquid,5 ml, twice a day, 12-month treatment', 'armGroupLabels': ['PXT3003 High dose']}, {'name': 'Placebo', 'type': 'OTHER', 'otherNames': ['Pleocompound PXT3003'], 'description': 'Liquid,5 ml, twice a day, 12-month treatment', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '59037', 'city': 'Lille', 'country': 'France', 'facility': 'Hôpital Roger Salengro', 'geoPoint': {'lat': 50.63391, 'lon': 3.05512}}, {'zip': '87042', 'city': 'Limoges', 'country': 'France', 'facility': 'CHU Dupuytren', 'geoPoint': {'lat': 45.83362, 'lon': 1.24759}}, {'zip': '69495', 'city': 'Lyon', 'country': 'France', 'facility': 'CHU Lyon Sud', 'geoPoint': {'lat': 45.74906, 'lon': 4.84789}}, {'zip': '13385', 'city': 'Marseille', 'country': 'France', 'facility': 'Hôpital La Timone', 'geoPoint': {'lat': 43.29695, 'lon': 5.38107}}, {'zip': '44093', 'city': 'Nantes', 'country': 'France', 'facility': 'Hôtel Dieu', 'geoPoint': {'lat': 47.21725, 'lon': -1.55336}}, {'zip': '75013', 'city': 'Paris', 'country': 'France', 'facility': 'Groupe Hospitalier Pitié-Salpétrière', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}], 'overallOfficials': [{'name': 'Shahram ATTARIAN, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'Hôpital La Timone'}, {'name': 'Viviane BERTRAND, PhD', 'role': 'STUDY_DIRECTOR', 'affiliation': 'Pharnext S.C.A.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Pharnext S.C.A.', 'class': 'OTHER'}, 'responsibleParty': {'type': 'SPONSOR'}}}}