NT-3 Levels and Function in Individuals With CMT

Z

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

Trial contacts and locations

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Central trial contact

Dawn Scott, RN

Data sourced from clinicaltrials.gov

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