Clinical trial NCT05011006: NT-3 Levels and Function in Individuals With CMT
Official title: Correlation of Circulating NT-3 Levels and Function in Individuals With Peripheral Neuropathy or Charcot-Marie-Tooth Neuropathy
Study type: OBSERVATIONAL
Overall status: UNKNOWN
Conditions: Charcot-Marie-Tooth Disease
Peripheral Neuropathy
Chronic Inflammatory Demyelinating Polyneuropathy
Keywords: NT-3
CMT1A
Neuropathy
Lead sponsor: Nationwide Children's Hospital
Sponsor class: OTHER
First posted: 2021-08-18
Enrollment: 50
Sex: ALL
Minimum age (years): 7
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male and female subjects, from 7 years of age and over with a definitive diagnosis of acquired peripheral neuropathy or genetically determined CMT neuropathy

Brief 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.

Eligibility criteria: 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

Primary outcomes:
measure: Assess change in Manual Muscle Testing (MMT)
timeFrame: Compare baseline to Year 1 and Year 2 visits.
measure: Assess change in 100 Meter Timed Test (100m):
timeFrame: Compare baseline to Year 1 and Year 2 visits.
measure: Assess change in Timed Functional Testing:
timeFrame: Compare baseline to Year 1 and Year 2 visits.
measure: Assess change in PROMIS Upper Extremity Function and Mobility Scales:
timeFrame: Compare baseline to Year 1 and Year 2 visits.
measure: Assess change in Abilities Captured Through Interactive Video Evaluation (ACTIVE)
timeFrame: Compare baseline to Year 1 and Year 2 visits.
measure: Assess change in CMT Pediatric Scale (CMTPedS)
timeFrame: Compare baseline to Year 1 and Year 2 visits.
measure: Assess change in Timed Up and Go
timeFrame: Compare baseline to Year 1 and Year 2 visits.
measure: Assess change in Forced Vital Capacity
timeFrame: Compare baseline to Year 1 and Year 2 visits.
measure: Assess change in Exploratory Gait Evaluation
timeFrame: Compare baseline to Year 1 and Year 2 visits.
Secondary outcomes:
Other outcomes:
Locations:
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
Study officials:
affiliation: Nationwide Children's Hospital
name: Zarife Sahenk, MD., PhD.
role: PRINCIPAL_INVESTIGATOR
References: