Rare CNS Genetic Disorder Study Award
Global Clinical Trials is initiating a new Phase III study in pediatric population in Russia. This important project is the continuation of the existing collaboration between GCT and our long-term partner pharmaceutical company based in the U.S.
It is a double-blind, randomized, placebo-controlled study to be followed by a long-term open-label treatment. The trial will be conducted in children and young adults with a rare genetic mutation in the X-chromosome. GCT is responsible for managing all the centers in Russia, including the full scope of clinical operations, logistics services and regulatory support. The regulatory submission took place last week, as planned.
The investigational medicinal product has already been studied in more than 1,500 subjects, both adults and children. The studies have shown that it is safe and well-tolerated. Furthermore, the data from the previous Phase II trial, managed regionally by GCT, proves the efficacy of the IP. The majority of the patients qualified for the continued treatment demonstrated reduction in seizures, one of the major symptoms of the disease.
Updated: July 2, 2019