NT-3 Levels and Function in Individuals With CMT

NCT ID: NCT05011006

Last Updated: 2025-06-24

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

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Recruitment Status

NOT_YET_RECRUITING

Total Enrollment

50 participants

Study Classification

OBSERVATIONAL

Study Start Date

2027-11-13

Study Completion Date

2030-06-30

Brief Summary

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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

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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

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

Study Design

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Observational Model Type

COHORT

Study Time Perspective

PROSPECTIVE

Interventions

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NT3 blood draw

blood draw

Intervention Type DIAGNOSTIC_TEST

Eligibility Criteria

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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
Minimum Eligible Age

7 Years

Eligible Sex

ALL

Accepts Healthy Volunteers

No

Sponsors

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Zarife Sahenk

OTHER

Sponsor Role lead

Responsible Party

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Zarife Sahenk

Principal Investigator

Responsibility Role SPONSOR_INVESTIGATOR

Principal Investigators

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Zarife Sahenk, MD., PhD.

Role: PRINCIPAL_INVESTIGATOR

Nationwide Children's Hospital

Locations

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Nationwide Children's Hospital

Columbus, Ohio, United States

Site Status

Countries

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United States

Central Contacts

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Dawn Scott, RN

Role: CONTACT

614722-2715

Facility Contacts

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Dawn Scott

Role: primary

(614)7222715

Other Identifiers

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19-0353

Identifier Type: -

Identifier Source: org_study_id

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