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Trial record 32 of 337 for:    Charcot Marie Tooth

Change in MUNIX in Patients With CMT1A Undergoing a Home Ankle Strengthening Program Versus Standard of Care

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ClinicalTrials.gov Identifier: NCT03715283
Recruitment Status : Recruiting
First Posted : October 23, 2018
Last Update Posted : February 15, 2019
Sponsor:
Information provided by (Responsible Party):
Ryan Castoro, Vanderbilt University Medical Center

Brief Summary:
Here the investigators aim to show that a focused lower extremity resistance strength training program in patients with Charcot-Marie-Tooth disease (CMT) results in increased motor strength of ankle plantar- and dorsi-flexion. The investigators will use motor unit index MUNIX and hand held dynamometry to correlate strength changes. The investigators believe that increased strength will correlate with an increased motor unit number and as such will prove that axonal renervation or improved recruitment is possible with a focused exercises in patients with CMT. Additionally, the investigators will show that that MUNIX declines over a 12-week period in patients with CMT whom continue standard of care. This will identify MUNIX as a responsive marker for disease progression in addition to detecting functional improvement, which will be valuable for future clinical trials.

Condition or disease Intervention/treatment Phase
Charcot-Marie-Tooth Disease Other: Home Ankle Strengthing Program Other: Standard of Care Not Applicable

  Show Detailed Description

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 50 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double (Investigator, Outcomes Assessor)
Primary Purpose: Diagnostic
Official Title: A Randomized Double Blind Longitudinal Study to Determine Motor Unit Number Index Variability in CMT1A Patients Undergoing a Home Ankle Strengthening Program Versus Standard of Care
Actual Study Start Date : January 15, 2019
Estimated Primary Completion Date : April 1, 2019
Estimated Study Completion Date : June 1, 2019


Arm Intervention/treatment
Experimental: Home Lower Extremity Strengthening
Patients in this arm will undergo a 12 week strengthening program which focuses on ankle dorsi- and plantar- flexion. Clinical visits will occur at baseline, 6 weeks and 12 weeks from the start of study. At each visit all patients will undergo a clinical exam, answer questionaires and undergo MUNIX testing.
Other: Home Ankle Strengthing Program
Patients in this arm will be given a USB or DVD video with description of ankle dorsi- and plantar- flexion resistance band exercise. They will also be given an outlined plan for there exercises, progression and an exercise diary. In general patients will start with a low resistance and low reps, over the 4 weeks they will increase the repetitions at the same resistance band. After the 4 weeks they will have the option to escalate the band resistance or continue at the same resistance and repetitions until comfortable progressing. The same 4 week progression will be used for each resistance band over the 12 weeks.

Experimental: No intervention
In this portion of the study patients will not be given any intervention. Clinical visits will occur at baseline, 6 weeks and 12 weeks from the start of study. At each visit all patients will undergo a clinical exam, answer questionaires and undergo MUNIX testing of both legs.
Other: Standard of Care
Patients in this arm will be directed to continue there standard care program. They will be asked to refrain from resistance exercise for 12 weeks.




Primary Outcome Measures :
  1. Change in motor unit number index (MUNIX) of the peroneal nerve in the treatment arm versus the untreated arm from baseline to 12 weeks (Peroneal Munix at 12 weeks - Peroneal Munix at baseline,0-1,000a.u., higher numbers represent more motor units) [ Time Frame: 12 weeks ]
  2. Change in motor unit number index (MUNIX) of the tibial nerve in the treatment arm versus the untreated arm from baseline to 12 weeks (Tibial MUNIX.at 12 weeks - Tibial MUNIX at baseline, 0-1000 arbitrary units, higher numbers represent more motor units) [ Time Frame: 12 weeks ]

Secondary Outcome Measures :
  1. Change in peroneal nerve motor unit number index (MUNIX) at 6 weeks between treatment arms (MUNIX at 6 weeks - MUNIX at Baseline; 0-1000 arbitrary units, higher numbers represent more motor units). [ Time Frame: 6 weeks ]
  2. Change in tibial nerve compound muscle action potential (CMAP) at 6 weeks between treatment arms ((Tibial CMAP at 6 weeks - Tibial CMAP at baseline (mV) 0-300mV, mV= milliVolts, higher numbers represent more motor units). [ Time Frame: 6 weeks ]
  3. Change in tibial nerve compound muscle action potential (CMAP) at 12 weeks between treatment arms (Tibial CMAP at 12 weeks - Tibial CMAP at baseline (mV) 0-300mV, mV= milliVolts, higher numbers represent more motor units). [ Time Frame: 12 weeks ]
  4. Change in peroneal nerve compound muscle action potential (CMAP) at 6 weeks between treatment arms (Peroneal CMAP at 6 weeks - Peroneal CMAP at baseline 0-300mV, mV = milliVolts, higher numbers represent more motor units). [ Time Frame: 6 weeks ]
  5. Change in peroneal nerve compound muscle action potential (CMAP) at 12 weeks between treatment arms (Peroneal CMAP at 12 weeks - Peroneal CMAP at baseline 0-300mV, mV= milliVolts, higher numbers represent more motor units). [ Time Frame: 12 weeks ]
  6. Change in muscle strength as measured by dynamometry of ankle dorsiflexion at 6 weeks between treatment arms (dorsiflexion strength at 6 weeks - dorsiflexion strength at baseline 0- 500 N, N=Newton, higher numbers represent means greater strength). [ Time Frame: 6 weeks ]
  7. Change in muscle strength as measured by dynamometry of ankle dorsiflexion at 12 weeks between treatment arms (dorsiflexion strength at 12 weeks - dorsiflexion strength at baseline 0-500 N, N=Newton, higher numbers represent means greater strength). [ Time Frame: 12weeks ]
  8. Change in muscle strength as measured by dynamometry of ankle plantarflexion at 6 weeks between treatment arms (plantarflexion strength at 6 weeks - plantarflexion strength at baseline 0-500N, N= Newton, higher numbers represent means greater strength). [ Time Frame: 6 weeks ]
  9. Change in muscle strength as measured by dynamometry of ankle plantarflexion at 12 weeks between treatment arms (plantarflexion strength at 12 weeks - plantarflexion strength at baseline 0-500N, N= newton higher numbers represent means greater strength). [ Time Frame: 12 weeks ]
  10. Change in the 6 minute walk test (6MWT) at 6 weeks between treatment arms (6MWT at 6 weeks - 6MWT at baseline 0-5,000m; m = meter, longer distances represent improved functional mobility). [ Time Frame: 6 weeks ]
  11. Change in the 6 minute walk test (6MWT) at 12 weeks between treatment arms (6MWT at 12 weeks - 6MWT at baseline 0-5,000m; m = meter, longer distances represent improved functional mobility). [ Time Frame: 12 weeks ]
  12. Change in Charcot Marie Tooth Disease neuropathy score (CMTNS) at 12 weeks between treatment arms (CMTNS at 12 weeks - CMTNS at baseline 0-36 arbitrary units, higher numbers represent more severe disease). [ Time Frame: 12 weeks ]
  13. Change in Charcot Marie Tooth Disease neuropathy score (CMTNS) at 6 weeks between treatment arms (CMTNS at 6 weeks - CMTNS at baseline 0-36 arbitrary units, higher numbers represent more severe disease). [ Time Frame: 6 weeks ]
  14. Change in peroneal nerve motor unit size index (MUSIX) at 6 weeks between treatment arms (Peroneal MUSIX at 6 weeks - Peroneal MUSIX at Baseline 0-1,000 arbitrary units, higher number represents more motor units). [ Time Frame: 6 weeks ]
  15. Change in peroneal nerve motor unit size index (MUSIX) at 12 weeks between treatment arms (Peroneal MUSIX at 12 weeks - Peroneal MUSIX at Baseline 0-1,000 arbitrary units, higher number represents more motor units). [ Time Frame: 12 weeks ]
  16. Change in tibial nerve motor unit size index (MUSIX) at 6 weeks between treatment arms (Tibial MUSIX at 6 weeks - Tibial MUSIX at Baseline 0-1,000 arbitrary units, higher number represents more motor units). [ Time Frame: 6 weeks ]
  17. Change in tibial nerve motor unit size index (MUSIX) at 12 weeks between treatment arms (Tibial MUSIX at 12 weeks - Tibial MUSIX at Baseline 0-1,000 arbitrary units, higher number represents more motor units). [ Time Frame: 12 weeks ]
  18. Change in tibial nerve motor unit number index (MUNIX) at 6 weeks between treatment arms (MUNIX at 6 weeks - MUNIX at Baseline; 0-1000 arbitrary units, higher number represents more motor units). [ Time Frame: 6 weeks ]


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Inclusion Criteria:

  • Adult Patients 18 years or older with genetically confirmed CMT1A
  • Adult Patients 18 years or older with a CMT1A genetically confirmed relative and a positive clinical exam or nerve conduction study consistent with CMT1A.
  • Unaffected persons 18 years or older with no past medical history of peripheral neuropathy

Exclusion Criteria:

  • Patients with a history of medical diseases that affect peripheral nerve function including diabetic neuropathy, uncontrolled thyroid dysfunction, amyloidosis, monoclonal gammopathy of uncertain significance or untreated vitamin deficiencies.
  • Patients with a history of other neurologic disease which may affect peripheral nerve function or extremity strength or function including stroke, seizures with a history of Todd's paralysis, Parkinson's Disease, Dementia, Guillen-Barre Syndrome, Myasthenia Gravis, Lambert Eaton Myasthenia Gravis or hypothyroidism.
  • Patients with ankle dorsiflexion strength of less than 3/5 in either limb on Medical Research Council scale.
  • Patients enrolled in a clinical trial (excluding natural history studies) in the past 12 months.
  • Patients who have undergone intense physical therapy, meaning more than 1 time per week for 6 or more weeks within the last 12 months.
  • Patient who do resistance training of the lower extremity more than 2 times per week for more than 3 months.
  • Patient in whom exercise would be consider dangerous including autonomic failure, postural orthostatic tachycardic syndrome (POTS), lower extremity deep vein thrombosis or pulmonary embolism, arterial insufficiency, uncontrolled pulmonary hypertension, uncontrolled hypertension or uncontrolled heart failure with reduced ejection fraction.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03715283


Contacts
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Contact: Ryan J Castoro, D.O., M.S. 6158356903 ryan.j.castoro@vumc.org
Contact: Stacy M Stark, M.D. stacy.m.stark@vumc.org

Locations
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United States, Tennessee
Vanderbilt University Medical Center Recruiting
Nashville, Tennessee, United States, 37212
Contact: Ryan Castoro, D.O.       ryan.j.castoro@vumc.org   
Sponsors and Collaborators
Ryan Castoro
Investigators
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Principal Investigator: Ryan J Castoro, D.O. Vanderbilt University Medical Center

Publications:
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Responsible Party: Ryan Castoro, Resident Physician, Vanderbilt University Medical Center
ClinicalTrials.gov Identifier: NCT03715283     History of Changes
Other Study ID Numbers: VR52854
First Posted: October 23, 2018    Key Record Dates
Last Update Posted: February 15, 2019
Last Verified: February 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Ryan Castoro, Vanderbilt University Medical Center:
MUNIX
Additional relevant MeSH terms:
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Tooth Diseases
Charcot-Marie-Tooth Disease
Nerve Compression Syndromes
Hereditary Sensory and Motor Neuropathy
Stomatognathic Diseases
Nervous System Malformations
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Neurodegenerative Diseases
Polyneuropathies
Peripheral Nervous System Diseases
Neuromuscular Diseases
Congenital Abnormalities
Genetic Diseases, Inborn