Ignite Creation Date:
2025-12-24 @ 9:22 PM
Ignite Modification Date:
2025-12-25 @ 7:08 PM
Study NCT ID:
NCT01289704
Status:
UNKNOWN
Last Update Posted:
2011-02-04
First Post:
2011-01-24
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D002607', 'term': 'Charcot-Marie-Tooth Disease'}], 'ancestors': [{'id': 'D015417', 'term': 'Hereditary Sensory and Motor Neuropathy'}, {'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'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'SINGLE', 'whoMasked': ['OUTCOMES_ASSESSOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 92}}, 'statusModule': {'overallStatus': 'UNKNOWN', 'lastKnownStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2011-02'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2010-10', 'completionDateStruct': {'date': '2012-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2011-02-03', 'studyFirstSubmitDate': '2011-01-24', 'studyFirstSubmitQcDate': '2011-02-03', 'lastUpdatePostDateStruct': {'date': '2011-02-04', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2011-02-04', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2011-10', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Walking ability of patients will be evaluated as the time needed to walk for 10 meters at normal speed for the patients', 'timeFrame': 'Baseline: 1 day before the rehabilitative protocol starts (T1)', 'description': 'The walking ability of patients will be evaluated through the "10 meters timed walk test" which will be performed at baseline (T1 or day-1), at the end of the three months of treatment (T2 or day90) and at the end of the three months of follow up (T3 or day180).'}, {'measure': 'Walking ability of patients will be evaluated after finishing the treatment as the time needed to walk for 10 meters at normal speed for the patients', 'timeFrame': 'at the end of treatment: day 90 (T2)', 'description': 'The walking ability of patients will be evaluated through the "10 meters timed walk test" which will be performed at baseline (T1 or day-1), at the end of the three months of treatment (T2 or day90) and at the end of the three months of follow up (T3 or day180).'}, {'measure': 'Walking ability of patients will be evaluated after finishing the treatment as the time needed to walk for 10 meters at normal speed for the patients', 'timeFrame': 'at the end of follow up: day 180 (T3)', 'description': 'The walking ability of patients will be evaluated through the "10 meters timed walk test" which will be performed at baseline (T1 or day-1), at the end of the three months of treatment (T2 or day90) and at the end of the three months of follow up (T3 or day180).'}], 'secondaryOutcomes': [{'measure': 'Balance will be evaluated through the Berg Scale', 'timeFrame': 'Baseline: 1 day before the rehabilitative protocol starts', 'description': 'The Berg scale is a tool to evaluate balance and ranges from 0 to 36 points being 36 normal'}, {'measure': 'Balance will be evaluated through the Berg Scale', 'timeFrame': 'at the end of treatment: day 90 (T2)', 'description': 'The Berg scale is a tool to evaluate balance and ranges from 0 to 36 points being 36 normal'}, {'measure': 'balance will be evaluted through the Berg scale', 'timeFrame': 'at the end of follow up: day 180 (T3)', 'description': 'The Berg scale is a tool to evaluate balance and ranges from 0 to 36 points being 36 normal'}, {'measure': 'Quality of life will be evaluated through the SF - 36 questionnaire', 'timeFrame': 'Baseline: 1 day before the rehabilitative protocol starts (T1)', 'description': 'The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index.'}, {'measure': 'Quality of life will be evaluated through the SF - 36 questionnaire', 'timeFrame': 'at the end of treatment: day 90 (T2)', 'description': 'The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index.'}, {'measure': 'Quality of life will be evaluated through the SF - 36 questionnaire', 'timeFrame': 'at the end of follow up: day 180 (T3)', 'description': 'The SF-36 is a multi-purpose, short-form health survey with only 36 questions. It yields an 8-scale profile of functional health and well-being scores as well as psychometrically-based physical and mental health summary measures and a preference-based health utility index.'}]}, 'oversightModule': {'oversightHasDmc': False}, 'conditionsModule': {'keywords': ['Charcot-Marie-Tooth disease,', 'CMT1A,', 'rehabilitation,', 'treadmill,', 'stretching,', 'proprioception,', 'Randomised Controlled Trial.'], 'conditions': ['Charcot-Marie-Tooth Disease', 'Charcot-Marie-Tooth Disease Type 1A']}, 'referencesModule': {'references': [{'pmid': '15610704', 'type': 'BACKGROUND', 'citation': 'Grandis M, Shy ME. Current Therapy for Charcot-Marie-Tooth Disease. Curr Treat Options Neurol. 2005 Jan;7(1):23-31. doi: 10.1007/s11940-005-0003-5.'}, {'pmid': '18254090', 'type': 'BACKGROUND', 'citation': 'Young P, De Jonghe P, Stogbauer F, Butterfass-Bahloul T. Treatment for Charcot-Marie-Tooth disease. Cochrane Database Syst Rev. 2008 Jan 23;2008(1):CD006052. doi: 10.1002/14651858.CD006052.pub2.'}, {'pmid': '15034573', 'type': 'BACKGROUND', 'citation': 'Passage E, Norreel JC, Noack-Fraissignes P, Sanguedolce V, Pizant J, Thirion X, Robaglia-Schlupp A, Pellissier JF, Fontes M. Ascorbic acid treatment corrects the phenotype of a mouse model of Charcot-Marie-Tooth disease. Nat Med. 2004 Apr;10(4):396-401. doi: 10.1038/nm1023. Epub 2004 Mar 21.'}, {'pmid': '17029975', 'type': 'BACKGROUND', 'citation': 'Pareyson D, Schenone A, Fabrizi GM, Santoro L, Padua L, Quattrone A, Vita G, Gemignani F, Visioli F, Solari A; CMT-TRIAAL Group. A multicenter, randomized, double-blind, placebo-controlled trial of long-term ascorbic acid treatment in Charcot-Marie-Tooth disease type 1A (CMT-TRIAAL): the study protocol [EudraCT no.: 2006-000032-27]. Pharmacol Res. 2006 Dec;54(6):436-41. doi: 10.1016/j.phrs.2006.09.001. Epub 2006 Sep 9.'}, {'pmid': '17060310', 'type': 'BACKGROUND', 'citation': 'Carter GT, Han JJ, Mayadev A, Weiss MD. Modafinil reduces fatigue in Charcot-Marie-Tooth disease type 1A: a case series. Am J Hosp Palliat Care. 2006 Oct-Nov;23(5):412-6. doi: 10.1177/1049909106292169.'}, {'pmid': '8328892', 'type': 'BACKGROUND', 'citation': 'Aitkens SG, McCrory MA, Kilmer DD, Bernauer EM. Moderate resistance exercise program: its effect in slowly progressive neuromuscular disease. Arch Phys Med Rehabil. 1993 Jul;74(7):711-5. doi: 10.1016/0003-9993(93)90031-5.'}, {'pmid': '8185450', 'type': 'BACKGROUND', 'citation': 'Kilmer DD, McCrory MA, Wright NC, Aitkens SG, Bernauer EM. The effect of a high resistance exercise program in slowly progressive neuromuscular disease. Arch Phys Med Rehabil. 1994 May;75(5):560-3.'}, {'pmid': '19197566', 'type': 'BACKGROUND', 'citation': 'van Pomeren M, Selles RW, van Ginneken BT, Schreuders TA, Janssen WG, Stam HJ. The hypothesis of overwork weakness in Charcot-Marie-Tooth: a critical evaluation. J Rehabil Med. 2009 Jan;41(1):32-4. doi: 10.2340/16501977-0274.'}, {'pmid': '7576421', 'type': 'BACKGROUND', 'citation': 'Carter GT, Abresch RT, Fowler WM Jr, Johnson ER, Kilmer DD, McDonald CM. Profiles of neuromuscular diseases. Hereditary motor and sensory neuropathy, types I and II. Am J Phys Med Rehabil. 1995 Sep-Oct;74(5 Suppl):S140-9. doi: 10.1097/00002060-199509001-00008.'}, {'pmid': '1565114', 'type': 'BACKGROUND', 'citation': 'Carter GT, Kilmer DD, Bonekat HW, Lieberman JS, Fowler WM Jr. Evaluation of phrenic nerve and pulmonary function in hereditary motor and sensory neuropathy, type I. Muscle Nerve. 1992 Apr;15(4):459-62. doi: 10.1002/mus.880150407.'}, {'pmid': '17443532', 'type': 'BACKGROUND', 'citation': 'Sackley C, Disler PB, Turner-Stokes L, Wade DT. Rehabilitation interventions for foot drop in neuromuscular disease. Cochrane Database Syst Rev. 2007 Apr 18;(2):CD003908. doi: 10.1002/14651858.CD003908.pub2.'}, {'pmid': '17964785', 'type': 'BACKGROUND', 'citation': 'Solari A, Laura M, Salsano E, Radice D, Pareyson D; CMT-TRIAAL Study Group. Reliability of clinical outcome measures in Charcot-Marie-Tooth disease. Neuromuscul Disord. 2008 Jan;18(1):19-26. doi: 10.1016/j.nmd.2007.09.006. Epub 2007 Oct 26.'}, {'pmid': '6513764', 'type': 'BACKGROUND', 'citation': 'Florence JM, Hagberg JM. Effect of training on the exercise responses of neuromuscular disease patients. Med Sci Sports Exerc. 1984 Oct;16(5):460-5. doi: 10.1249/00005768-198410000-00007.'}, {'pmid': '2654435', 'type': 'BACKGROUND', 'citation': 'Jones DR, Speier J, Canine K, Owen R, Stull GA. Cardiorespiratory responses to aerobic training by patients with postpoliomyelitis sequelae. JAMA. 1989 Jun 9;261(22):3255-8.'}, {'pmid': '15895353', 'type': 'BACKGROUND', 'citation': 'Aitkens S, Kilmer DD, Wright NC, McCrory MA. Metabolic syndrome in neuromuscular disease. Arch Phys Med Rehabil. 2005 May;86(5):1030-6. doi: 10.1016/j.apmr.2004.09.012.'}, {'pmid': '11254787', 'type': 'BACKGROUND', 'citation': 'Pfeiffer G, Wicklein EM, Ratusinski T, Schmitt L, Kunze K. Disability and quality of life in Charcot-Marie-Tooth disease type 1. J Neurol Neurosurg Psychiatry. 2001 Apr;70(4):548-50. doi: 10.1136/jnnp.70.4.548.'}, {'pmid': '16186535', 'type': 'BACKGROUND', 'citation': 'Vinci P, Serrao M, Millul A, Deidda A, De Santis F, Capici S, Martini D, Pierelli F, Santilli V. Quality of life in patients with Charcot-Marie-Tooth disease. Neurology. 2005 Sep 27;65(6):922-4. doi: 10.1212/01.wnl.0000176062.44360.49.'}, {'pmid': '15824348', 'type': 'BACKGROUND', 'citation': 'Shy ME, Blake J, Krajewski K, Fuerst DR, Laura M, Hahn AF, Li J, Lewis RA, Reilly M. Reliability and validity of the CMT neuropathy score as a measure of disability. Neurology. 2005 Apr 12;64(7):1209-14. doi: 10.1212/01.WNL.0000156517.00615.A3.'}, {'pmid': '16574732', 'type': 'BACKGROUND', 'citation': 'Graham RC, Hughes RA. Clinimetric properties of a walking scale in peripheral neuropathy. J Neurol Neurosurg Psychiatry. 2006 Aug;77(8):977-9. doi: 10.1136/jnnp.2005.081497. Epub 2006 Mar 30.'}, {'pmid': '12091180', 'type': 'BACKGROUND', 'citation': 'ATS Committee on Proficiency Standards for Clinical Pulmonary Function Laboratories. ATS statement: guidelines for the six-minute walk test. Am J Respir Crit Care Med. 2002 Jul 1;166(1):111-7. doi: 10.1164/ajrccm.166.1.at1102. No abstract available.'}, {'pmid': '5772056', 'type': 'BACKGROUND', 'citation': 'Black LF, Hyatt RE. Maximal respiratory pressures: normal values and relationship to age and sex. Am Rev Respir Dis. 1969 May;99(5):696-702. doi: 10.1164/arrd.1969.99.5.696. No abstract available.'}, {'pmid': '7792547', 'type': 'BACKGROUND', 'citation': 'Berg K, Wood-Dauphinee S, Williams JI. The Balance Scale: reliability assessment with elderly residents and patients with an acute stroke. Scand J Rehabil Med. 1995 Mar;27(1):27-36.'}], 'seeAlsoLinks': [{'url': 'http://www.acmt-rete.it', 'label': 'Acmt-Rete is a non-profit organization made by a group of patients to help the CMT community and to promote research in the field of hereditary neuropathies'}]}, 'descriptionModule': {'briefSummary': 'Charcot-Marie-Tooth neuropathy type 1A (CMT1A) is one of the most common inherited neurological disorders. The study will evaluate the efficacy and safety of an innovative rehabilitation protocol constituted by exercises at the treadmill and by a stretching and proprioceptive approach. A total of 92 patients will be enrolled in the study and treated in a controlled, randomized, single blind, way. To recruit a high number of patients with CMT1A the study will be multicentric and will comprehend four of the most active clinical centers in the field of CMT, in Italy. People with CMT1A are very motivated in entering rehabilitation protocols because to date there is no effective therapy for this disease. Therefore, the investigators expect a high compliance by the patients. With the present project the investigators plan to clarify several unanswered questions: 1) the efficacy and safety of treadmill over a more conventional protocol; 2) the duration and frequency of any rehabilitation treatment; 3) the most sensitive outcome measures to evaluate the efficacy of rehabilitation approach in patients with CMT.', 'detailedDescription': 'A multicentre, prospective, randomised, controlled, single blind study to evaluate the impact of aerobic exercise, based on a tightly controlled program at the treadmill, on the rehabilitation therapy of CMT 1A neuropathy.\n\nComparing aerobic training at the treadmill combined with respiratory physiotherapy, stretching and proprioceptive exercises (TreSPE- treated group) with a more conventional treatment only composed by respiratory physiotherapy, stretching and proprioceptive exercises (SPE- control group) will provide information on the impact of Treadmill in CMT1A.\n\n92 patients (23 per centre) will be enrolled and randomly assigned to TreSPE (n = 46) or to SPE (n = 46). Both groups will be treated for three months and followed up for six months.\n\nNo serious side effects are expected with TreSPE, as also suggested by our preliminary results. For safety reasons blood pressure (BP), heart rate (HR) and an electrocardiogram will be recorded during the rehabilitation treatment when the treating physician considers it necessary. The patients will be allowed, if needed, to hold at the parallel bars of the treadmill during exercise. According to the American Thoracic Society (ATS) guidelines the cardiopulmonary exercise test will be interrupted if BP raises at 240/120 and/or HR to 220-patients age.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '70 Years', 'minimumAge': '18 Years', 'healthyVolunteers': True, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Clinical diagnosis of CMT1A\n* Genetic confirmation (17p112 chromosome duplication)\n* Age 18 - 70 years old\n* Ability to accomplish the primary outcome measure (10 meter walking test) without support, with or without ankle foot orthoses (AFO)\n* Ability to walk on a treadmill on a horizontal plane for 20 minutes at a speed of 1.5 km/h with or without support at the bars\n* Score at the Mobility Scale between 2 and 11\n* Signed written informed consent to participate\n\nExclusion Criteria:\n\n* Diagnosis of Hereditary Neuropathy with Liability to Pressure Palsies (HNPP) or any other type of CMT\n* Other associated causes of neuropathy\n* Vestibular affections, psychiatric, cardiovascular and lung disorders or severe arthropathic changes in the lower limbs\n* Non ambulating patients or patients always requiring even monolateral support to walk\n* Other neurological disorders'}, 'identificationModule': {'nctId': 'NCT01289704', 'acronym': 'TreSPE', 'briefTitle': 'Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A)', 'organization': {'class': 'OTHER', 'fullName': 'University of Genova'}, 'officialTitle': 'A Multicenter Study to Evaluate the Effects on Charcot-Marie-Tooth Neuropathy Type 1A of a Composite Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program.', 'orgStudyIdInfo': {'id': 'GUP09013'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'TreSPE', 'description': 'Treatment with treadmill, proprioceptive and stretching exercises', 'interventionNames': ['Other: TreSPE']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'SPE', 'description': 'Proprioceptive and stretching exercises', 'interventionNames': ['Other: TreSPE', 'Other: SPE']}], 'interventions': [{'name': 'TreSPE', 'type': 'OTHER', 'otherNames': ['SPE'], 'description': "1. treadmill: 5'warm up, 20'aerobic exercise (gradually incremented to 30', from session to session, if possible), 5'warm down, controlling hearth rate, blood pressure and SaO2.\n2. Respiratory Physiotherapy: positive Expiratory Pressure (PEP) bottle for 10' (in various postures), and postural training, for the other 10'.\n3. Stretching for 20', of the triceps surae, tibialis posterior, extensor and flexors digitorum longus and brevis, both at the bed and in a static position.\n4. Proprioceptive and postural kinesitherapy according to the Perfetti method.\n5. Balance Exercising consists of exercises carried on by basculating bars with improving difficulties in the instruments utilized and in the tasks with therapist supervision and near a handbar preventing falls.", 'armGroupLabels': ['SPE', 'TreSPE']}, {'name': 'SPE', 'type': 'OTHER', 'description': "1. Respiratory Physiotherapy for 20', consisting of Positive Expiratory Pressure (PEP) bottle for 10' (in various postures), and postural training according to the Mèzières technique, for the other 10'.\n2. Stretching for 20', of the triceps surae, tibialis posterior, extensor and flexors longus and brevis, both at the bed and in a static position.\n3. Proprioceptive and postural kinesitherapy according to the neurocognitive method.\n4. Balance Exercising consists of exercises carried on by moving bars with improving difficulties in the instruments utilized and in the tasks with therapist supervision and near a handlebar preventing falls.", 'armGroupLabels': ['SPE']}]}, 'contactsLocationsModule': {'locations': [{'zip': '16132', 'city': 'Genoa', 'country': 'Italy', 'contacts': [{'name': 'Angelo Schenone, Professor', 'role': 'CONTACT', 'email': 'aschenone@neurologia.unige.it', 'phone': '+39 010 353 7040'}], 'facility': 'University of Genoa', 'geoPoint': {'lat': 44.40478, 'lon': 8.94439}}, {'zip': '20133', 'city': 'Milan', 'country': 'Italy', 'contacts': [{'name': 'Davide Pareyson, MD', 'role': 'CONTACT', 'email': 'dpareyson@istituto-besta.it', 'phone': '+39 02 23943001'}], 'facility': 'I.R.C.C.S. Foundation, Besta Institute', 'geoPoint': {'lat': 45.46427, 'lon': 9.18951}}, {'zip': '00194', 'city': 'Rome', 'country': 'Italy', 'contacts': [{'name': 'Luca Padua, MD', 'role': 'CONTACT', 'email': 'lpadua@rm.unicatt.it', 'phone': '+39 06 33086231'}], 'facility': 'Don Carlo Gnocchi Foundation', 'geoPoint': {'lat': 41.89193, 'lon': 12.51133}}, {'zip': '37134', 'city': 'Verona', 'country': 'Italy', 'contacts': [{'name': 'Gian Maria Fabrizi, MD', 'role': 'CONTACT', 'email': 'gianmaria.fabrizi@univr.it', 'phone': '+39 045 8124461'}], 'facility': 'Departement of Neurological and Visual Sciences, University of Verona', 'geoPoint': {'lat': 45.43854, 'lon': 10.9938}}], 'centralContacts': [{'name': 'Margherita A Monti Bragadin, MD', 'role': 'CONTACT', 'email': 'margherita.montibragadin@gmail.com', 'phone': '0039-010-3537040'}], 'overallOfficials': [{'name': 'Angelo E Schenone, MD', 'role': 'PRINCIPAL_INVESTIGATOR', 'affiliation': 'University of Genova'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'University of Genova', 'class': 'OTHER'}, 'responsibleParty': {'oldNameTitle': 'Gianluigi Mancardi', 'oldOrganization': 'Neuroscience, Ophtalmology and Genetic Departement'}}}}