An Open Observational Study of Clinical and Electrophysiological Outcomes in Male and Female Patients With CMT Type 1 & 2, and Aged-matched Healthy Controls

Status: Completed
Location: See all (2) locations...
Study Type: Observational

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.

Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: Accepts Healthy Volunteers

• 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

• Healthy male or female volunteers

• Age ≥18 years

• Able to give written informed consent

United States
Ohio State University
Other Locations
Aarhus University Hospital
Time Frame
Start Date: April 7, 2021
Completion Date: March 3, 2022
Target number of participants: 30
CMT Patients
Individuals with Charcot-Marie-Tooth Disease Types 1 and 2.
Healthy Controls
Healthy age-matched volunteers
Kristina Kelly, William D Arnold, Helga H Lillevang, Henning Andersen, Hatice Tankisi
Collaborators: Aarhus University Hospital, Ohio State University
Leads: NMD Pharma A/S

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