NT-3 Levels and Function in Individuals With CMT

Trial statusNot yet recruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age7+
SponsorZarife Sahenk

About this trial

This study will assess the serum NT-3 levels in individuals with the diagnosis of peripheral neuropathy or any type of Charcot-Marie-Tooth Neuropathy (CMT) and correlate this with function.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Males or females from 7 years of age or older

Diagnosis of acquired peripheral neuropathy (PN) such as due to diabetes, chemo-induced, autoimmune chronic inflammatory demyelinating polyneuropathy (CIDP) established by a report of electrical studies or with a known genetic CMT diagnosis. Definitive diagnosis of PN is established with a report of electrical studies, EMG/nerve conduction studies.2

Perform assessments to the best of their ability with reliable results as deemed by the evaluator.

Ability to attend scheduled appointments

Disqualifiers

Current pregnancy per medical history

Has a medical condition or extenuating circumstances that, in the opinion of the investigator, might compromise the subject's wellbeing, safety, or clinical interpretability

Trial population

Male and female subjects, from 7 years of age and over with a definitive diagnosis of acquired peripheral neuropathy or genetically determined CMT neuropathy

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • NT3 blood draw

    Diagnostic test

    blood draw

Treatment groups

No treatment groups listed

Trial outcomes

Primary outcomes

1

Assess change in Manual Muscle Testing (MMT)

Weakness in the major motor groups can be documented by using manual muscle testing. This exam allows the symmetry of strength, along with distribution and degree of weakness to be assessed. The Medical Research Council lists the major motor groups as: neck flexion, hip extension, knee flexion, ankle plantar flexion, and hip abduction. Depending on the clinical circumstances, the exam can be individualized. The Medical Research Council provides a scale ranging from 0-5. Inter-rater reliability will be documented by all evaluators of the trial. A rating of 5 indicates full range of motion, whereas a rating of 0 indicates no movement.

Time frame
Compare baseline to Year 1 and Year 2 visits.
2

Assess change in 100 Meter Timed Test (100m):

Directions for study procedures will be provided in a way the subject can understand (i.e. verbal and/or written instruction, demonstration, practice, etc.). Once the subject is comfortable with study procedures, they will complete the 100m test one time. Patients will be asked to run a set course of 25 meters for 4 laps for a total distance of 100 meters. The time to complete in seconds will be recorded.

Time frame
Compare baseline to Year 1 and Year 2 visits.
3

Assess change in Timed Functional Testing:

Timed Functional Testing can include time to ascend/descend 4 standard-sized stairs, time to rise from the floor (from NSAD), and the 10-meter run (from the NSAD).

Time frame
Compare baseline to Year 1 and Year 2 visits.
4

Assess change in PROMIS Upper Extremity Function and Mobility Scales:

The PROMIS Upper Extremity Function and Mobility instruments are patient-reported outcomes (PRO) that utilize a patient reported and/or parent proxy scale based on the patient's age that measures an individual's perceived upper extremity function and mobility.

Time frame
Compare baseline to Year 1 and Year 2 visits.

Secondary outcomes

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Zarife Sahenk

Lead sponsor

Nationwide Children's Hospital

Sponsor institution

This trial is not recruiting at the moment. You can still explore other options: