Clinical trials in Charcot-Marie-Tooth disorders: a retrospective and preclinical assessment.

ABSTRACT:

Objective

This study aimed to evaluate the progression of clinical and preclinical trials in Charcot-Marie-Tooth (CMT) disorders.

Background

CMT has historically been managed symptomatically and with genetic counseling. The evolution of molecular and pathologic understanding holds a therapeutic promise in gene-targeted therapies.

Methods

ClinicalTrials.gov from December 1999 to June 2022 was data extracted for CMT with preclinical animal gene therapy trials also reviewed by PubMed search.

Results

The number of active trials was 1 in 1999 and 286 in 2022. Academic settings accounted for 91% and pharmaceutical companies 9%. Of the pharmaceutical and academic trials, 38% and 28%, respectively, were controlled, randomized, and double-blinded. Thirty-two countries p

SUBMITTER: Nair MA 

PROVIDER: S-EPMC10556688 | biostudies-literature | 2023

REPOSITORIES: biostudies-literature