NT-3 Levels and Function in Individuals With CMT

Last updated: June 18, 2025
Sponsor: Zarife Sahenk
Overall Status: Active - Not Recruiting

Phase

N/A

Condition

Peripheral Neuropathy

Neuropathy

Neurologic Disorders

Treatment

NT3 blood draw

Clinical Study ID

NCT05011006
19-0353
  • Ages > 7
  • All Genders

Study 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.

Eligibility Criteria

Inclusion

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 bythe evaluator.

  • Ability to attend scheduled appointments

  • Ability to provide informed consent (or assent for ages 9-18)

Exclusion

Exclusion Criteria:

  • Current pregnancy per medical history

  • Has a medical condition or extenuating circumstances that, in the opinion of theinvestigator, might compromise the subject's wellbeing, safety, or clinicalinterpretability

Study Design

Total Participants: 50
Treatment Group(s): 1
Primary Treatment: NT3 blood draw
Phase:
Study Start date:
November 13, 2027
Estimated Completion Date:
June 30, 2030

Study 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.

Connect with a study center

  • Nationwide Children's Hospital

    Columbus, Ohio 43205
    United States

    Site Not Available

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