Correlation of Circulating NT-3 Levels and Function in Individuals With Peripheral Neuropathy or Charcot-Marie-Tooth Neuropathy
Zarife Sahenk
Summary
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.
Description
This study will assess the serum NT-3 levels in those with a peripheral neuropathy diagnosis which will help to determine the therapeutic levels of NT-3 in subjects that will receive AAV1.NT-3 gene therapy in a future study. This study also aims to define the natural history of any type of Charcot-Marie Tooth Disease (CMT), better define the rate of disease progression and skeletal muscle involvement. Another aim is to generate a registry of well-characterized CMT patients who may be candidates for future trials.
Eligibility
- Age range
- 7+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: * 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 * Ability to provide informed con…
Interventions
- Diagnostic TestNT3 blood draw
blood draw
Location
- Nationwide Children's HospitalColumbus, Ohio