Charcot-Marie-Tooth Disease Clinical Trial
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
Verified date | December 2019 |
Source | Vanderbilt University Medical Center |
Contact | n/a |
Is FDA regulated | No |
Health authority | |
Study type | Interventional |
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.
Status | Completed |
Enrollment | 19 |
Est. completion date | September 1, 2019 |
Est. primary completion date | September 1, 2019 |
Accepts healthy volunteers | Accepts Healthy Volunteers |
Gender | All |
Age group | 18 Years and older |
Eligibility |
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. |
Country | Name | City | State |
---|---|---|---|
United States | Vanderbilt University Medical Center | Nashville | Tennessee |
Lead Sponsor | Collaborator |
---|---|
Vanderbilt University Medical Center |
United States,
Bas J, Delmont E, Fatehi F, Salort-Campana E, Verschueren A, Pouget J, Lefebvre MN, Grapperon AM, Attarian S. Motor unit number index correlates with disability in Charcot-Marie-Tooth disease. Clin Neurophysiol. 2018 Jul;129(7):1390-1396. doi: 10.1016/j.clinph.2018.04.359. Epub 2018 Apr 16. — View Citation
Li J. Inherited neuropathies. Semin Neurol. 2012 Jul;32(3):204-14. doi: 10.1055/s-0032-1329198. Epub 2012 Nov 1. Review. — View Citation
Nandedkar SD, Barkhaus PE, Stålberg EV. Motor unit number index (MUNIX): principle, method, and findings in healthy subjects and in patients with motor neuron disease. Muscle Nerve. 2010 Nov;42(5):798-807. doi: 10.1002/mus.21824. — View Citation
Type | Measure | Description | Time frame | Safety issue |
---|---|---|---|---|
Primary | 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) | 12 weeks | ||
Primary | 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) | 12 weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 12 weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 12 weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 12weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 12 weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 12 weeks | ||
Secondary | 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). | 12 weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 12 weeks | ||
Secondary | 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). | 6 weeks | ||
Secondary | 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). | 12 weeks | ||
Secondary | 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). | 6 weeks |
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