NT-3 Levels and Function in Individuals With CMT

Zarife Sahenk

Status: Enrolling

Conditions: Peripheral Neuropathy; Chronic Inflammatory Demyelinating Polyneuropathy; Charcot-Marie-Tooth Disease

Treatments: Diagnostic Test: NT3 blood draw

Study type: Observational

Funder types: Other

Identifiers

NCT05011006

19-0353

Details and patient eligibility

About

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.

Full 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.

Enrollment: 50 estimated patients

Sex: All

Ages: 7+ years old

Volunteers: No Healthy Volunteers

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 consent (or assent for ages 9-18)

Exclusion criteria

• 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