Charcot-Marie-Tooth DiseasePhase 2/3TreSPENCT01289704
Treadmill, Stretching and Proprioceptive Exercise (TreSPE) Rehabilitation Program for Charcot-Marie-Tooth Neuropathy Type 1A (CMT1A)
A Phase 2/3 study testing TreSPE and SPE in people with Charcot-Marie-Tooth Disease and Charcot-Marie-Tooth Disease Type 1A, run by University of Genova at 4 sites in Italy.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 18 to 70 yearsHealthy volunteers can take part
- Conditions the study is for
- Charcot-Marie-Tooth Disease, Charcot-Marie-Tooth Disease Type 1A
- What is being tested?
- TreSPE and SPE
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 92 participants
- Where
- 4 sitesItaly
- Study dates
- Feb 2011to Jan 2012
- Phase
- Phase 2/3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 70 years
- All sexes
- Healthy volunteers accepted
- Adult, Older adult
Who may be able to join
- Clinical diagnosis of CMT1A
- Genetic confirmation (17p112 chromosome duplication)
- Age 18 - 70 years old
- Ability to accomplish the primary outcome measure (10 meter walking test) without support, with or without ankle foot orthoses (AFO)
- 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
- Score at the Mobility Scale between 2 and 11
- Signed written informed consent to participate
Who cannot join
- Diagnosis of Hereditary Neuropathy with Liability to Pressure Palsies (HNPP) or any other type of CMT
- Other associated causes of neuropathy
- Vestibular affections, psychiatric, cardiovascular and lung disorders or severe arthropathic changes in the lower limbs
- Non ambulating patients or patients always requiring even monolateral support to walk
- Other neurological disorders
Trial locations and contacts
4 sites in 1 country.
Italy4 sites
University of Genoa
Genoa, 16132
Angelo Schenone, Professor, +39 010 353 7040, aschenone@neurologia.unige.it
I.R.C.C.S. Foundation, Besta Institute
Milan, 20133
Davide Pareyson, MD, +39 02 23943001, dpareyson@istituto-besta.it
Don Carlo Gnocchi Foundation
Rome, 00194
Luca Padua, MD, +39 06 33086231, lpadua@rm.unicatt.it
Departement of Neurological and Visual Sciences, University of Verona
Verona, 37134
Gian Maria Fabrizi, MD, +39 045 8124461, gianmaria.fabrizi@univr.it
About this study
Official title: 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.
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.
Read the detailed description
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.
Comparing 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.
92 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.
No 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.
- Being tested
- TreSPE, SPE
- Keywords
- Charcot-Marie-Tooth disease,, CMT1A,, rehabilitation,, treadmill,, stretching,, proprioception,, Randomised Controlled Trial.
- Medical subject headings
- Charcot-Marie-Tooth Disease
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
TreSPEOther
Also called SPE
- 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.
- Respiratory Physiotherapy: positive Expiratory Pressure (PEP) bottle for 10' (in various postures), and postural training, for the other 10'.
- 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.
- Proprioceptive and postural kinesitherapy according to the Perfetti method.
- 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.
SPEOther
- 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'.
- Stretching for 20', of the triceps surae, tibialis posterior, extensor and flexors longus and brevis, both at the bed and in a static position.
- Proprioceptive and postural kinesitherapy according to the neurocognitive method.
- 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.
| Study group | Role | What they receive |
|---|---|---|
| TreSPE | Experimental | Treatment with treadmill, proprioceptive and stretching exercises Receives: Other: TreSPE |
| SPE | Active comparator | Proprioceptive and stretching exercises Receives: Other: TreSPE; Other: SPE |
What is being measured
The primary outcome is the main question the study is designed to answer.
Walking ability of patients will be evaluated as the time needed to walk for 10 meters at normal speed for the patients
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).
Measured: Baseline: 1 day before the rehabilitative protocol starts (T1)
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
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).
Measured: at the end of treatment: day 90 (T2)
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
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).
Measured: at the end of follow up: day 180 (T3)
Secondary outcomes (6)
Balance will be evaluated through the Berg Scale
The Berg scale is a tool to evaluate balance and ranges from 0 to 36 points being 36 normal
Measured: Baseline: 1 day before the rehabilitative protocol starts
Balance will be evaluated through the Berg Scale
The Berg scale is a tool to evaluate balance and ranges from 0 to 36 points being 36 normal
Measured: at the end of treatment: day 90 (T2)
balance will be evaluted through the Berg scale
The Berg scale is a tool to evaluate balance and ranges from 0 to 36 points being 36 normal
Measured: at the end of follow up: day 180 (T3)
Quality of life will be evaluated through the SF - 36 questionnaire
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.
Measured: Baseline: 1 day before the rehabilitative protocol starts (T1)
Quality of life will be evaluated through the SF - 36 questionnaire
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.
Measured: at the end of treatment: day 90 (T2)
Quality of life will be evaluated through the SF - 36 questionnaire
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.
Measured: at the end of follow up: day 180 (T3)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Feb 2011 (estimated)
- Study startFeb 2011
- Primary completionOct 2011 (estimated)
- Study completionJan 2012 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (22)Papers the sponsor has linked to this study.
- Other trials for Charcot-Marie-Tooth DiseaseStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2/3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 92 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University of Genova
- Sponsor type
- University, hospital or other
- Investigators
- Angelo E Schenone, MD, Principal investigator, University of Genova
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01289704
- Sponsor's protocol ID
- GUP09013
- Acronym
- TreSPE
- Status last verified
- Oct 2010
- First submitted
- 24 Jan 2011
- Last updated
- 4 Feb 2011
- Results posted
- No
- Data monitoring committee
- No
Publications
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.
PubMed 15610704Young 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.
PubMed 18254090Passage 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.
PubMed 15034573Pareyson 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.
PubMed 17029975Carter 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.
PubMed 17060310Aitkens 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.
PubMed 8328892Kilmer 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.
PubMed 8185450van 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.
PubMed 19197566Carter 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.
PubMed 7576421Carter 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.
PubMed 1565114Sackley 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.
PubMed 17443532Solari 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.
PubMed 17964785Florence 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.
PubMed 6513764Jones 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.
PubMed 2654435Aitkens 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.
PubMed 15895353
Study at a glance
From the registry record, last updated by the sponsor on 4 Feb 2011. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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.
- ClinicalTrials.gov ID
- NCT01289704
- Acronym / sponsor ID
- TreSPE / GUP09013
- Keywords
- Charcot-Marie-Tooth disease,, CMT1A,, rehabilitation,, treadmill,, stretching,, proprioception,, Randomised Controlled Trial.
- Phase
- Phase 2/3
- Study type
- Interventional
- Recruitment status
- Unknown status
- Enrolment
- 92 participants (estimated)
- Lead sponsor
- University of GenovaUniversity, hospital or other
- Study start
- Feb 2011
- Primary completion
- Oct 2011 (estimated)
- Study completion
- Jan 2012 (estimated)
- First posted
- 4 Feb 2011 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
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.
Comparing 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.
92 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.
No 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| TreSPE | Experimental | Other: TreSPETreatment with treadmill, proprioceptive and stretching exercises |
| SPE | Active comparator | Other: TreSPE, Other: SPEProprioceptive and stretching exercises |
Who may qualify
- Ages
- 18 to 70 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- Clinical diagnosis of CMT1A
- Genetic confirmation (17p112 chromosome duplication)
- Age 18 - 70 years old
- Ability to accomplish the primary outcome measure (10 meter walking test) without support, with or without ankle foot orthoses (AFO)
- 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
Key exclusion criteria (5 listed)
- Diagnosis of Hereditary Neuropathy with Liability to Pressure Palsies (HNPP) or any other type of CMT
- Other associated causes of neuropathy
- Vestibular affections, psychiatric, cardiovascular and lung disorders or severe arthropathic changes in the lower limbs
- Non ambulating patients or patients always requiring even monolateral support to walk
- Other neurological disorders
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| TreSPEAlso: SPE1. 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. 2. Respiratory Physiotherapy: positive Expiratory Pressure (PEP) bottle for 10' (in various postures), and postural training, for the other 10'. 3. 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. 4. Proprioceptive and postural kinesitherapy according to the Perfetti method. 5. 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. | Other | SPE, TreSPE |
| SPE1. 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'. 2. Stretching for 20', of the triceps surae, tibialis posterior, extensor and flexors longus and brevis, both at the bed and in a static position. 3. Proprioceptive and postural kinesitherapy according to the neurocognitive method. 4. 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. | Other | SPE |
Primary outcome
Walking ability of patients will be evaluated as the time needed to walk for 10 meters at normal speed for the patients
Time frame: Baseline: 1 day before the rehabilitative protocol starts (T1)
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).
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
Time frame: at the end of treatment: day 90 (T2)
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).
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
Time frame: at the end of follow up: day 180 (T3)
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).
6 secondary outcomes
Balance will be evaluated through the Berg Scale
Time frame: Baseline: 1 day before the rehabilitative protocol starts
Balance will be evaluated through the Berg Scale
Time frame: at the end of treatment: day 90 (T2)
balance will be evaluted through the Berg scale
Time frame: at the end of follow up: day 180 (T3)
Quality of life will be evaluated through the SF - 36 questionnaire
Time frame: Baseline: 1 day before the rehabilitative protocol starts (T1)
Quality of life will be evaluated through the SF - 36 questionnaire
Time frame: at the end of treatment: day 90 (T2)
Quality of life will be evaluated through the SF - 36 questionnaire
Time frame: at the end of follow up: day 180 (T3)
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 4 Feb 2011.
- Background: PubMed 15610704Grandis 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.
- Background: PubMed 18254090Young 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.
- Background: PubMed 15034573Passage 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.
- Background: PubMed 17029975Pareyson 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.
- Background: PubMed 17060310Carter 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.
- Background: PubMed 8328892Aitkens 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.
- Background: PubMed 8185450Kilmer 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.
- Background: PubMed 19197566van 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.
- Background: PubMed 7576421Carter 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.
- Background: PubMed 1565114Carter 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.
- Background: PubMed 17443532Sackley 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.
- Background: PubMed 17964785Solari 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.
- 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 neuropathiesLink provided by the sponsor.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Angelo E Schenone, MD principal investigator
University of Genova
4 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2/3 study testing TreSPE and SPE in people with Charcot-Marie-Tooth Disease and Charcot-Marie-Tooth Disease Type 1A, run by University of Genova at 4 sites in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 5 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 4 Feb 2011. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 4 sites in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Margherita A Monti Bragadin, MD (0039-010-3537040, margherita.montibragadin@gmail.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Walking ability of patients will be evaluated as the time needed to walk for 10 meters at normal speed for the patients (time frame: Baseline: 1 day before the rehabilitative protocol starts (T1)). The study lists 3 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT01289704, which also lists 22 linked publications.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 4 Feb 2011.