A survey of registered pharmacological clinical trials on rare neurological diseases in children in 2010-2020.

ABSTRACT:

Objective

To clarify the current state of methodology of clinical trials for rare neurological diseases in children, and to provide a basis for the further optimization of the trial design.

Methods

Data of clinical trials for the rare neurological diseases with childhood onset (searched through https://rarediseases.info.nih.gov/diseases and www.Orpha.net) registered on the Clinicaltrils.gov from January 2010 to June 2020 was collected. Analysis on the methodology of the clinical trials were performed, focusing on initiator of the studies, multi or single research center, study design, sample size, and the endpoint using in the trial.

Results

A total of 162 clinical trials were included, covering only 7.3% (61/835) of rare neurological diseases in children. 101 (62.3%)

SUBMITTER: Chang X 

PROVIDER: S-EPMC9672510 | biostudies-literature | 2022

REPOSITORIES: biostudies-literature