NCT05011006

NT-3 Levels and Function in Individuals With CMT

Correlation of Circulating NT-3 Levels and Function in Individuals With Peripheral Neuropathy or Charcot-Marie-Tooth Neuropathy

• Status: Not Yet Recruiting
• Phase: —
• Study type: Observational
• Enrollment: 50 (estimated)
• Sponsor: Zarife Sahenk · Academic / Other
• Sex: All
• Age: 7 Years
• Healthy volunteers: Not accepted

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.

Detailed 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.

Conditions

• Charcot-Marie-Tooth Disease
• Peripheral Neuropathy
• Chronic Inflammatory Demyelinating Polyneuropathy

Interventions

Type	Name	Description
DIAGNOSTIC_TEST	NT3 blood draw	blood draw

Timeline

• Start date: 2027-11-13
• Primary completion: 2030-06-01
• Completion: 2030-06-01
• First posted: 2021-08-18
• Last updated: 2025-06-24

Locations

1 site across 1 country: United States