Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls (NCT04980807) | Clinical Trial Compass
CompletedNot Applicable
Observational Study of Neuromuscular Function in CMT Type 1&2 and Healthy Controls
United States, Denmark30 participantsStarted 2021-04-07
Plain-language summary
The primary aim of this study is to discover whether patients with CMT type 1 and 2 have neuromuscular junction transmission deficits.
The secondary aim is to collect information of different clinical and electrophysiological test procedures to perform possible future clinical trials in CMT patients.
Who can participate
Age range
18 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria for CMT Patients
* Age ≥ 18 years
* Diagnosis of CMT confirmed by:
* Clinical presentation and electro diagnostics or genetics
* Physical Features (all must apply): 1) Ambulation for at least 10 meters, without a brace 2) Left and right ankle plantar flexion Medical Research Council (MRC) grade 2 to 5, inclusive 3) Left and right ankle dorsiflexion MRC manual muscle testing (MMT) grade 2 to 4+, inclusive.
* Ability to adhere to the study visit schedule/procedures, and to understand and comply with protocol requirements
* Stable concomitant medications for 2 months prior to enrolment
* Signed written informed consent
Exclusion Criteria for CMT Patients
* Severe deformity or ankle contracture that would sufficiently limit passive range of motion to affect assessment of dorsiflexion strength
* Ulceration that would interfere with functional ability
* Recent major surgery
* Significant change in physical activity or exercise (e.g., significant increase or decrease in intensity or frequency) within 8 weeks before Study Day 1 or inability to maintain the baseline level of physical activity throughout the study
* Any disability or condition that would prevent completion of the study tasks
* Moderate to severe Neuropathic or inflammatory/musculoskeletal pain that would interfere with performance of the tests
* A diagnosis of a primary NMJ disorder such as myasthenia gravis, Lambert Eaton Myasthenic Syndrome or congenital myasthenic disorder
Inclusion Criteria f…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
Repetitive Nerve Stimulation - difference between CMT patients and healthy controls
Timeframe: Baseline
2
Repetitive Nerve Stimulation - difference between CMT patients and healthy controls
Timeframe: Baseline
3
Single Fiber Electro Myography (sfEMG) - difference between CMT patients and healthy controls
Timeframe: Baseline
4
Single Fiber Electro Myography (sfEMG) - difference between CMT patients and healthy controls